Quantcast

Subscribe to:
POZ magazine
E-newsletters
Join POZ: Facebook MySpace Twitter Pinterest
Tumblr Google+ Flickr MySpace
POZ Personals
Sign In / Join
Username:
Password:
Welcome, Guest. Please login or register.
July 22, 2014, 07:28:28 AM

Login with username, password and session length


Members
Stats
  • Total Posts: 631419
  • Total Topics: 47801
  • Online Today: 218
  • Online Ever: 585
  • (January 07, 2014, 02:31:47 PM)
Users Online

Welcome


Welcome to the POZ/AIDSmeds Community Forums, a round-the-clock discussion area for people with HIV/AIDS, their friends/family/caregivers, and others concerned about HIV/AIDS.  Click on the links below to browse our various forums; scroll down for a glance at the most recent posts; or join in the conversation yourself by registering on the left side of this page.

Privacy Warning:  Please realize that these forums are open to all, and are fully searchable via Google and other search engines. If you are HIV positive and disclose this in our forums, then it is almost the same thing as telling the whole world (or at least the World Wide Web). If this concerns you, then do not use a username or avatar that are self-identifying in any way. We do not allow the deletion of anything you post in these forums, so think before you post.

  • The information shared in these forums, by moderators and members, is designed to complement, not replace, the relationship between an individual and his/her own physician.

  • All members of these forums are, by default, not considered to be licensed medical providers. If otherwise, users must clearly define themselves as such.

  • Forums members must behave at all times with respect and honesty. Posting guidelines, including time-out and banning policies, have been established by the moderators of these forums. Click here for “Am I Infected?” posting guidelines. Click here for posting guidelines pertaining to all other POZ/AIDSmeds community forums.

  • We ask all forums members to provide references for health/medical/scientific information they provide, when it is not a personal experience being discussed. Please provide hyperlinks with full URLs or full citations of published works not available via the Internet. Additionally, all forums members must post information which are true and correct to their knowledge.

  • Product advertisement—including links; banners; editorial content; and clinical trial, study or survey participation—is strictly prohibited by forums members unless permission has been secured from POZ.

To change forums navigation language settings, click here (members only), Register now

Para cambiar sus preferencias de los foros en español, haz clic aquí (sólo miembros), Regístrate ahora

Finished Reading This? You can collapse this or any other box on this page by clicking the symbol in each box.

Author Topic: I ♥ Gene Therapy  (Read 12130 times)

0 Members and 1 Guest are viewing this topic.

Offline freewillie99

  • Member
  • Posts: 304
I ♥ Gene Therapy
« on: May 30, 2009, 02:01:51 PM »
Not sure whether this is the work done at UCLA which was previously reported on.  Anyone?

http://www.prlog.org/10245717-asgt-12th-annual-meeting-gene-therapy-controls-hiv-research-shows.html

ASGT 12th Annual Meeting: Gene therapy controls HIV, research shows

Stem cells containing protective genes may provide permanent control of human immunodeficiency virus (HIV) in infected individuals, according to research presented at the American Society of Gene Therapy 12th Annual Meeting, May 30.

PRLog (Press Release) – May 30, 2009 – SAN DIEGO - Stem cells containing protective genes may provide permanent control of human immunodeficiency virus (HIV) in infected individuals, according to research presented at the American Society of Gene Therapy 12th Annual Meeting, May 30.

In a landmark study, researchers demonstrated that a hematopoietic stem cell therapy could safely and effectively provide long-lasting control of HIV.

“This study was the first phase II randomized, controlled, double-blind study for cell-delivered HIV gene therapy and the first controlled HIV gene therapy study to show positive impact on viral load and CD4 count,” said Geoff Symonds, PhD, senior research director at Calimmune, Inc. “This study is a major advance in the field and it paves the way for future treatment with this new therapeutic paradigm.”

Seventy-four patients were enrolled in the study’s two treatment arms. There were no serious adverse events reported from the gene therapy.

An estimated 1 million Americans and 35 million individuals worldwide, are infected with HIV, the virus that causes AIDS. Conventional HIV treatments, such as highly active antiretroviral therapy (HAART), are effective at slowing the disease’s progression by controlling the amount of virus in the body. These therapies, however, require a lifelong regimen of medications and are hindered by severe side effects and immense costs.

The American Society of Gene Therapy (ASGT) 12th Annual Meeting is the world’s largest scientific meeting surrounding the latest developments in gene and cell therapy, attended by nearly 2,000 researchers from around the world and featuring 60 scientific presentations.

ASGT is a non-profit medical and professional organization representing researchers and scientists devoted to the discovery of new gene and cell therapies. Established in 1996, ASGT is the largest association of individuals involved in gene and cell therapeutics with approximately 2,500 members in the United States and worldwide. Additional information is available at www.asgt.org.

« Last Edit: May 30, 2009, 02:49:23 PM by freewillie99 »
Beware Romanians bearing strange gifts

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #1 on: May 30, 2009, 02:16:11 PM »
This does sound so promising, gene therapy in general, and this article in particular.

I assume they altered the CCR5 mutation? They also don't define "long-lasting" control. For how long did the study go for? Basically a lot of details left out, would be great to find more info. about this study with 74 people. I wonder where we can find more specific information about this study? I guess maybe on their website if one is a member?

Thanks for posting ;)
« Last Edit: May 30, 2009, 02:19:26 PM by Inchlingblue »

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #2 on: May 30, 2009, 02:47:28 PM »

I assume they altered the CCR5 mutation?


This is all I could find with a quick search:

"Calimmune, Inc. (Tucson, Arizona) is developing a stem cell HIV/AIDS therapy that blocks the expression of the CCR5 co-receptor on T lymphocytes. After Dr. David Baltimore (President Emeritus of Caltech/Nobel Laureate) and Dr. Irvin Chen (Director of the UCLA AIDS Institute) established proof-of-concept and animal safety/efficacy data, Calimmune is now taking this innovative therapy to the clinic."
Beware Romanians bearing strange gifts

Offline simpleguy

  • Member
  • Posts: 67
Re: I ♥ Gene Therapy
« Reply #3 on: May 30, 2009, 03:59:02 PM »
I found an abstract on Nature about the clinical trial.

http://www.nature.com/nm/journal/v15/n3/full/nm.1932.html

Gene transfer has potential as a once-only treatment that reduces viral load, preserves the immune system and avoids lifetime highly active antiretroviral therapy. This study, which is to our knowledge the first randomized, double-blind, placebo-controlled, phase 2 cell-delivered gene transfer clinical trial, was conducted in 74 HIV-1–infected adults who received a tat-vpr–specific anti-HIV ribozyme (OZ1) or placebo delivered in autologous CD34+ hematopoietic progenitor cells. There were no OZ1-related adverse events. There was no statistically significant difference in viral load between the OZ1 and placebo group at the primary end point (average at weeks 47 and 48), but time-weighted areas under the curve from weeks 40–48 and 40–100 were significantly lower in the OZ1 group. Throughout the 100 weeks, CD4+ lymphocyte counts were higher in the OZ1 group. This study indicates that cell-delivered gene transfer is safe and biologically active in individuals with HIV and can be developed as a conventional therapeutic product.
« Last Edit: May 30, 2009, 04:01:49 PM by simpleguy »
2008 JUL: Sustiva OCT: Rayataz DEC: Kaletra • 2009 Off meds • 2011 Intelence • 2012+ Complera

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #4 on: June 05, 2009, 03:24:38 PM »
Deleted because posted in wrong thread by mistake.
« Last Edit: June 05, 2009, 06:29:35 PM by Inchlingblue »

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #5 on: June 08, 2009, 02:27:25 PM »
Not sure whether this is the work done at UCLA which was previously reported on.  Anyone?

The link from Simpleguy above refers to the study at UCLA that was reported in February. Whether that is the same study as this one isn't clear. I don't think so since the UCLA one is Mitsuyasu and this one is David Baltimore (Calimmune), unless they're working together.

And even though they are both gene therapy they seem to be using different approaches. Although they both mention 74 patients/subjects enrolled in study, which makes it confusing. Arrgghhh.
 
« Last Edit: June 08, 2009, 02:52:25 PM by Inchlingblue »

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #6 on: June 08, 2009, 03:01:23 PM »
The study is indeed incouraging but from the thread posted on February I report the following:

In an interview with AFP, Mitsuyasu said this experiment was a Phase II trial in the long, three-phase process to test prototype treatments for safety and effectiveness.  He said he would not put the technique to the final, third phase of the process. Instead, the team would learn from its experience, modify the technique and start again with tests on a smaller group of volunteers

This means it will take several years, perhaps a decade before we will see the final results. Dr. Frascino from thebody.com commented on this study:
http://www.thebody.com/Forums/AIDS/SafeSex/Current/Q201895.html

Please consider also that it is another addition to antiretrovirals and it will not cure.
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #7 on: June 08, 2009, 03:21:50 PM »
The study is indeed incouraging but from the thread posted on February I report the following:

In an interview with AFP, Mitsuyasu said this experiment was a Phase II trial in the long, three-phase process to test prototype treatments for safety and effectiveness.  He said he would not put the technique to the final, third phase of the process. Instead, the team would learn from its experience, modify the technique and start again with tests on a smaller group of volunteers

This means it will take several years, perhaps a decade before we will see the final results. Dr. Frascino from thebody.com commented on this study:
http://www.thebody.com/Forums/AIDS/SafeSex/Current/Q201895.html

Please consider also that it is another addition to antiretrovirals and it will not cure.


But the study reported by the OP in this particular thread is from a company called Calimmune and it may not be the same one as the one you are referring to. The approaches look to be different, the Calimmune study blocks the CCR5 receptor.

This would not be a cure for everyone but if someone with CCR5-tropic virus can have their immune system successfully re-jigged by way of gene therapy then they would for all intents and purposes be genetically resistant and be cured of HIV without the need for HAART.

I'm not saying it's as easy as pie to achieve this but there is enough proof-of-concept evidence to indicate that this is indeed very possible and, in fact, likely to happen at some point.
« Last Edit: June 08, 2009, 04:30:12 PM by Inchlingblue »

Offline elf

  • Member
  • Posts: 596
Re: I ♥ Gene Therapy
« Reply #8 on: June 10, 2009, 01:16:36 AM »
Gene therapy has been awaited for decades, but there are still no results where needed: cystic fibrosis and HIV...
Let's have a Kiki!

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #9 on: June 10, 2009, 01:48:14 AM »
Gene therapy has been awaited for decades, but there are still no results where needed: cystic fibrosis and HIV...

During the 8 years of the Bush administration progress was very slow due to the limitations on stem cell research.

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #10 on: June 10, 2009, 08:41:13 AM »
Gene therapy has been awaited for decades, but there are still no results where needed: cystic fibrosis and HIV...

Elf,

What's your point?  The first experimental gene therapy treatment was only performed in 1990.  I realize that one of the attributes of America in the 21st century is a sense of entitlement and desire for instant gratification, but science moves at it's own pace.  This is incredibly complicated stuff and not going to happen overnight.  
« Last Edit: June 10, 2009, 09:28:43 AM by freewillie99 »
Beware Romanians bearing strange gifts

Offline mousey

  • Member
  • Posts: 22
Re: I ♥ Gene Therapy
« Reply #11 on: June 10, 2009, 02:08:08 PM »
Nothing can really catch my attention when it reaches phase III and beyond.
But I really hope that someone somewhere in the planet can find a cure or at least to control it and stops the spread of this virus. Though we are now able to control it, the costs and time is really unbearable.



:: Believe in a cure ::

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #12 on: June 10, 2009, 08:34:44 PM »
Nothing can really catch my attention when it reaches phase III and beyond.
But I really hope that someone somewhere in the planet can find a cure or at least to control it and stops the spread of this virus. Though we are now able to control it, the costs and time is really unbearable.





The issue is that we have currently nothing promising in phase III. My concern is that most people puts too much entusiasm and hope in a research or study that at the end will lead to nowhere. The disappoint would hit heavy in such case.
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #13 on: June 11, 2009, 03:10:56 PM »
Jumping genes fight HIV

Feb. 19, 2008

UCL (University College London) scientists have taken a significant step in understanding how retroviruses such as HIV can move between species and the biological mechanisms behind the ‘jumping genes' which make some monkeys immune. They will now use this knowledge to develop a gene therapy treatment for HIV/AIDS in humans.

The international team of researchers, coordinated by Professor Greg Towers, UCL Infection and Immunity, and funded by the Wellcome Trust, have identified a combination of genes in a species of monkey that protects against retroviruses - a particularly opportunistic family of viruses that can integrate into the host's genome and replicate as part of the cell's DNA. The team's findings are published today in Proceedings of the National Academy of Sciences (PNAS).

Professor Towers explained: "HIV causes AIDS and affects around 40 million people worldwide. Research has shown that HIV entered the human population from a chimpanzee retrovirus called SIV early in the 20th century. In order for a virus to successfully cross the species barrier and jump into a new species, it first has to bypass the new host's innate immune system, mediated by a combination of genes and proteins. One such gene, called TRIM5, has been shown to protect certain species from retroviruses - but unfortunately the human TRIM5 gene does not protect against HIV infection."

The team found that a species of Asian monkey called Rhesus Macaques have a sophisticated ‘antiviral arsenal' that can protect them against retroviruses. By closely examining TRIM5 in this species, they demonstrate that in some monkeys another gene called Cyclophilin has been joined to the TRIM5 gene, generating a TRIMCyp fusion.

Dr Sam Wilson, the paper's first author, said: "Cyclophilin is very good at grabbing viruses as they enter cells. By fusing Cyclophilin to TRIM5, a gene is made that is good at grabbing viruses and good at destroying them. This is the second time that this fusion has been identified - a TRIMCyp gene also exists in South American Owl Monkeys and, until now, this was thought to be an evolutionary one-off.

"This new research shows that a TRIMCyp has evolved independently in two separate species - it's like lightening has struck twice. It's a remarkable example of convergent evolution, where organisms independently evolve similar traits as a result of having to adapt to similar environments. It also highlights the evolutionary selection pressure that viruses like HIV can apply."

Professor Greg Towers explained further: "The discovery is a compelling example of how ‘jumping genes' can shuffle an organism's genetic makeup, generating useful new genes, and it is an exciting possibility for novel treatments for HIV/AIDS.

"About 25 per cent of Rhesus Macaques have the TRIM5 and a TRIMCyp gene, greatly expanding their antiviral arsenal. The others have an immunity, based around TRIM5, that protects them against a different combination of viruses. The gene seems to be evolving to protect the individual species from a range of different virus sequences."

Professor Towers and his team now aim to develop humanised TRIMCyp that blocks HIV infection by artificially fusing human Cyclophilin and human TRIM5. Professor Towers said: "We can then introduce the TRIMCyp into stem cells, using gene therapy technologies, and the stem cells could repopulate the patient with blood cells that are immune to HIV. This work, already underway, could offer a real possibility of novel treatments for HIV/AIDS."

LINK:

http://www.scientistlive.com/European-Science-News/Genetics/Jumping_genes_fight_HIV/19810/

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #14 on: June 11, 2009, 09:15:43 PM »
Jumping genes fight HIV

Jumping gene flash it's a gas gas gas!
Beware Romanians bearing strange gifts

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #15 on: June 11, 2009, 10:40:12 PM »
Jumping gene flash it's a gas gas gas!

You silly LOL 

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #16 on: June 15, 2009, 01:05:18 PM »
Even though this study was not directly with HIV, it could have implications for the future success of gene therapy of all diseases:

Promising stem cell-gene therapy in genetics

A study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology. The study, published in the May 31, 2009 early online edition of Nature, is a major milestone on the path from the laboratory to the clinic.

"It's been ten years since human stem cells were first cultured in a Petri dish," says the study's leader Juan-Carlos Izpisúa Belmonte, Ph.D., a professor in the Gene Expression Laboratory and director of the Center of Regenerative Medicine in Barcelona (CMRB), Spain. "The hope in the field has always been that we'll be able to correct a disease genetically and then make iPS cells that differentiate into the type of tissue where the disease is manifested and bring it to clinic."

Although several studies have demonstrated the efficacy of the approach in mice, its feasibility in humans had not been established. The Salk study offers the first proof that this technology can work in human cells.

Belmonte's team, working with Salk colleague Inder Verma, Ph.D., a professor in the Laboratory of Genetics, and colleagues at the CMRB, and the CIEMAT in Madrid, Spain, decided to focus on Fanconi anaemia (FA), a genetic disorder responsible for a series of hematological abnormalities that impair the body's ability to fight infection, deliver oxygen, and clot blood. Caused by mutations in one of 13 Fanconi anaemia (FA) genes, the disease often leads to bone marrow failure, leukaemia, and other cancers. Even after receiving bone marrow transplants to correct the hematological problems, patients remain at high risk of developing cancer and other serious health conditions.

After taking hair or skin cells from patients with Fanconi anaemia, the investigators corrected the defective gene in the patients' cells using gene therapy techniques pioneered in Verma's laboratory. They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells using a combination of transcription factors, OCT4, SOX2, KLF4 and cMYC. The resulting FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.

Since bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells is the most prominent feature of Fanconi anaemia, the researchers then tested whether patient-specific iPS cells could be used as a source for transplantable hematopoietic stem cells. They found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

"We haven't cured a human being, but we have cured a cell," Belmonte explains. "In theory we could transplant it into a human and cure the disease."

Although hurdles still loom before that theory can become practise-in particular, preventing the reprogrammed cells from inducing tumours-in coming months Belmonte and Verma will be exploring ways to overcome that and other obstacles. In April 2009, they received a $6.6 million from the California Institute Regenerative Medicine (CIRM) to pursue research aimed at translating basic science into clinical cures.

"If we can demonstrate that a combined iPS-gene therapy approach works in humans, then there is no limit to what we can do," says Verma.

LINK:

http://www.scientistlive.com/European-Science-News/Genetics/Promising_stem_cell-gene_therapy_in_genetics/22505/

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #17 on: October 29, 2009, 01:33:17 PM »
Here is an update on the recent grant Chen's lab received. They will be working alongside Calimmune. 

Irvin Chen: HIV/AIDS

Chen, director of the UCLA AIDS Institute and a professor of microbiology, immunology and molecular genetics, received $19,999,580 to develop a method to block HIV infection and reproduction in the human body.
 
The need for novel approaches to the treatment of HIV infection has never been greater, according to Chen. New infections continue to occur in California, nationally and worldwide despite decades of prevention efforts. Additionally, the number of people living with HIV is rising due to improved management of their infection. As a result, California, which ranks second in the nation behind only New York in diagnosed cases of HIV infection, has identified 102,800 men, women and children who carry the virus. Estimates of the number of state residents who are infected but have yet to be diagnosed are as high as 33,500. Worldwide, it is estimated that 33 million people are living with HIV.
 
Chen's disease team will focus on RNA interference, a means to block the function of genes in the human body. He proposes that RNA interference can be used to block HIV-1 infection, a subtype of HIV, and its reproduction within the body. In this case, his team proposes blocking expression of CCR5, a key co-receptor used by HIV-1 to gain entry into host cells.
 
The approach is designed to mimic the effects of a naturally occurring mutation in humans that renders certain individuals resistant to HIV-1 infection. When RNA interference is introduced into a stem cell, its blocking activity will be present throughout the lifetime of the stem cell — theoretically, the lifespan of a human being — providing a lifelong therapy for HIV-1 infection.
 
"If you can artificially knock out CCR5, you can make the cells resistant to HIV," Chen said. "We thought we could use RNA interference — and we proved in preclinical and animal models that it works. Now we are ready to test this theory in human clinical trials."
 
In effect, researchers will be creating a new blood system that carries the RNA interference therapy in it. Chen said that, in theory, an effective stem cell RNA interference therapy will require only a single treatment, as opposed to the current lifetime administration of expensive anti-HIV-1 drugs, which often result in serious side effects.
 

"The goal of lifelong control or even, potentially, eradication of HIV via a stem cell therapy will not be straightforward," Chen said. "We have assembled the combined AIDS and stem cell expertise of investigators from four California academic institutions, as well as a corporate partner and a host of world-renowned advisers. Each will contribute unique expertise toward the development of a safe and efficacious therapeutic path into patients."
 
Independent reviewers of Chen's grant said the proposal was very strong and that "the resources and investigators are outstanding and the team is superb, both scientifically and in therapy development."

LINK:

http://www.newsroom.ucla.edu/portal/ucla/state-grants-totaling-49-19-million-111822.aspx
 

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #18 on: November 02, 2009, 08:42:26 AM »
This is incredibly promising stuff.  I've had some discussions with Dr. Baltimore and he's top notch.  Hopefully he and Dr. Chen can get this going in a timely manner.
Beware Romanians bearing strange gifts

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #19 on: February 25, 2010, 05:30:17 PM »
Gene-based stem cell therapy specifically removes cell receptor that attracts HIV

By Enrique Rivero February 25, 2010

FINDINGS:              
UCLA AIDS Institute researchers successfully removed CCR5 — a cell receptor to which HIV-1 binds for infection but which the human body does not need — from human cells. Individuals who naturally lack the CCR5 receptor have been found to be essentially resistant to HIV.
 
Using a humanized mouse model, the researchers transplanted a small RNA molecule known as short hairpin RNA (shRNA), which induced RNA interference into human blood stem cells to inhibit the expression of CCR5 in human immune cells.
 
IMPACT:
The findings provide evidence that this strategy can be an effective way to treat HIV-infected individuals, by prompting long-term and stable reduction of CCR5.
 
AUTHORS:
Saki Shimizu, Patrick Hong, Balamurugan Arumugam, Lauren Pokomo, Joshua Boyer, Naoya Koizumi, Panyamol Kittipongdaja, Angela Chen, Greg Bristol, Zoran Galic, Jerome A. Zack, Otto Yang, Irvin S.Y. Chen, Benhur Lee and Dong Sung An, all of UCLA.
 
JOURNAL:              
Blood, Journal of the American Society of Hematology
 
FUNDING:
The research was funded by a Rheumatology Fellowship Training grant, the UCLA AIDS Institute, the UCLA Center for AIDS Research, the National Institute of Allergy and Infectious Diseases, the National Heart, Lung and Blood Institute, and the National Cancer Institute.


LINK:

http://www.newsroom.ucla.edu/portal/ucla/gene-based-stem-cell-therapy-specifically-154045.aspx

Offline Cosmicdancer

  • Member
  • Posts: 145
Re: I ♥ Gene Therapy
« Reply #20 on: February 25, 2010, 05:46:53 PM »
That's great news, and they achieved this milestone just a few months after receiving a $20 million grant.  Given that this was achieved in mice with humanized immune systems, I'm wondering if they need to move onto primates before testing in humans. 
Summer, 2007 - &$#@?
November, 2007 - Tested poz, 300,000 vl, 560 cd4
Feb, 2008 - 57,000 vl, 520 cd4, started Atripla
June, 2008 - undetectable, 612 cd4
January, 2009 - undetectable, 670 cd4
May, 2009 - undetectable, 593 cd4
Sept, 2009 - 83 vl, 763 cd4, 34%
Dec, 2009 - undetectable, 889 cd4, 32%
April, 2010 - undetectable, 860 cd4, 31%
October, 2010 - undetectable, 800 cd4, 38%
April, 2011 - undetectable, t-cell test not done
October, 2011 - undetectable
April, 2012 - undetectable, 850 cd4, 39%
November, 2012 - undetectable, 901 cd4, 41%
April, 2013 - undetectable, 846 cd4, 36%
October, 2013 - undetectable
May, 2014 - undetectable, 784 cd4, 48%

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #21 on: April 14, 2010, 10:15:40 AM »
http://www.whittierdailynews.com/news/ci_14864878

City of Hope researchers say genetic therapy has potential to cure HIV

By Emma Gallegos, Staff Writer
Posted: 04/11/2010 06:39:01 PM PDT

DUARTE - Doctors can prescribe powerful new drugs that keep HIV in check, but researchers at the City of Hope want to knock it down with a cure.

No matter how many drugs a doctor prescribes, HIV lingers forever in a patient's system, like a smoldering fire that threatens to flare up if patient misses too many doses.

Genetic therapy has the potential to extinguish those lingering hot spots and ultimately cure patients of the virus, according to John Rossi, a researcher at the City of Hope.

Rossi is working on a pair of studies that could do just that. The first one is entering the second phase of human clinical trials, and the other has been tested on mice and is safe enough to be tested on humans.

HIV is devastating, because it attacks the immune system by destroying its key fighter: the T-cell. The first study tinkers with the genes inside T-cells so that they can resist HIV.

Researchers at the City of Hope created a strain of anti-HIV genes that they inserted into blood stem cells that will become T-cells.

These stem cells were injected into the four patients in the study two years ago.

The experiment has gone well and the next phase of the clinical trial will begin in a few weeks, Rossi said.

The City of Hope is working with researchers at Colorado State University on a study that tries to knock out the virus with a multi-pronged attack, Rossi said.

They've created molecules in a lab that prevents the virus from entering a cell. If the virus does manage to get in, these molecules can stop it from reproducing, according to Ramesh Akkina, a professor of pathology overseeing the research on mice in Colorado.

Although HIV treatments have made leaps and bounds over the last few decades, current treatments are far from ideal, Rossi said. The best case scenario is that HIV patients have to take their drugs every day of the rest of their lives to stay healthy.

In practice, it can be hard to convince people with HIV to take the drugs they need to survive, according to Jenny O'Malley, the registered nurse program manager at the AIDS Service Center in Pasadena.

A young woman who had been going to the agency died last week because she wasn't taking her drugs, O'Malley said.

Some of her patients don't take their drugs because they're in denial or the stigma is still strong. Other patients don't seem to grasp the importance of taking their drugs every day or they worry about side-effects.



Beware Romanians bearing strange gifts

Offline veritas

  • Member
  • Posts: 1,408
Re: I ♥ Gene Therapy
« Reply #22 on: April 14, 2010, 10:28:10 AM »

Free,

Nice find !!! With all the great research going on in many areas, we have a REAL race for the cure!

Who will be first?  Not that it matters, as long as it is done (after 30 yrs and god knows how many lives).

It's about time!

v

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #23 on: April 14, 2010, 01:32:56 PM »
What keeps me skeptical of a significant breakthrough in the near future is the fact that many experts are not convinced of that, claiming that it is quite unlikely. Two of them are Dr. Gallant, a famous HIV specialist, and Dr. Frascino from thebody.com forum.

Dr. Frascino is more involved in the research area and Dr. Gallant in the therapy field. Both continue to argue that a cure or a major archievement in HIV therapy is still in a distant future. How distant they obviously don't know but they don't seem too enthusiastic even with the current efforts and discoverings. Since everything is relative (Einstein's theory), the only hope is that distant don't mean a decade or more.
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #24 on: June 16, 2010, 02:51:06 PM »
The two articles linked below give a very good update on what's going on with some of the gene therapy/stem cell studies.

Triple-punch gene therapy targets HIV

LINK:

http://www.nature.com/news/2010/100616/full/news.2010.301.html

Gene Therapy Shows Promise for Blocking HIV, Controlling AIDS

LINK:

http://www.businessweek.com/news/2010-06-16/gene-therapy-shows-promise-for-blocking-hiv-controlling-aids.html

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #25 on: June 16, 2010, 03:33:44 PM »
Yes, I still ♥ Gene Therapy.  Xman, not so much.

Mark my words, something wonderful is going to come from the research.
Beware Romanians bearing strange gifts

Offline mercuryman

  • Member
  • Posts: 20
Re: I ♥ Gene Therapy
« Reply #26 on: June 16, 2010, 04:00:34 PM »
Inch,

Great articles! We need new avenues of treatment for sure! Know that your time, research, and dissemination of HIV treatment information is appreciated!!!

My hat goes off to "V" as well.........

May we all remain OPTOMISTIC/HOPEFUL........and be of encouragement to others.......

I count my blessings for each day of life.......

Latest CD4 count: 3
Latest Viral Load: 645,000

My best to all of you,

Mercuryman


Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #27 on: June 16, 2010, 04:06:05 PM »
Quotes regarding the same study but cut from a different article:

"It was a very carefully conducted study, and it's a proof of concept that it's doable," says Carl June, a medical oncologist who is conducting similar gene-therapy studies with HIV at the University of Pennsylvania School of Medicine. "Stem cells until now have been very resistant to this approach."

"June is more optimistic still. "Now it's just a problem of engineering, where we need to work out the details," June says. "I don't see any show stoppers."

Xman, with immediate effect, please get on the phone to Dr. June at Penn (he of Zinc Finger fame) and inform him that he doesn't know squat.

LINK :http://news.sciencemag.org/sciencenow/2010/06/can-gene-therapy-cure-aids.html

« Last Edit: June 16, 2010, 04:16:31 PM by freewillie99 »
Beware Romanians bearing strange gifts

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #28 on: June 16, 2010, 04:13:51 PM »
 Free: great link, thanks.

mercuryman, you're an inspiration ;)
« Last Edit: June 16, 2010, 04:25:09 PM by Inchlingblue »

Offline geobee

  • Member
  • Posts: 261
Re: I ♥ Gene Therapy
« Reply #29 on: June 16, 2010, 06:42:28 PM »
This is really cool... if they can crank up the number of modified cells, find a way to ablate the remaining cells safely -- heck this might just work.  I'm optimistic...

.... but I also appreciate the skeptics.  I hope we can all step back from complaining about each other.  We're all in this boat together.

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #30 on: June 16, 2010, 09:39:38 PM »
Please could somebody here explain this part:

The trial piggybacked on a standard treatment, in which individuals with AIDS receive transplants of their own previously saved blood stem cells, in an attempt to prevent the development of lymphoma (blood cancer).

Aren't stem cells infected too with the virus? What does they really mean with previously saved stem cells?
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #31 on: June 16, 2010, 09:45:10 PM »
...
« Last Edit: June 16, 2010, 10:03:56 PM by xman »
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #32 on: June 16, 2010, 09:53:16 PM »
If you read very carefully the second article you'll notice that a transplant and a shutdown of the bown marrow and immune system was made as for the german patient in Berlin. They also state that they're studying less toxic ways to follow such a procedure and to have the same outcome. Less toxic can meaning everything. This it what concerns me more. It could be still lifethreatening!
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #33 on: June 16, 2010, 10:10:58 PM »
Xman, with immediate effect, please get on the phone to Dr. June at Penn (he of Zinc Finger fame) and inform him that he doesn't know squat.

I would say he should contact Dr. Frascino for this issue.

Hi,

Yep, I do admit to being perhaps the most optimistic guy in the universe. However, I'm also a trained scientist and I follow HIV research very closely. I'm not a skeptic; I'm a realist. See below. Certainly a cure could result from some serendipitous discovery, but that remains highly unlikely. To sum up, I'm an optimistic realist!

Dr. Bob


http://www.thebody.com/Forums/AIDS/SafeSex/Current/Q209193.html

But perhaps he is missinformed or too pessimistic (he indeed claims to be an optimistic realist, a little different than veritas who is an optimistic dreamer). What do you think?
« Last Edit: June 16, 2010, 10:16:10 PM by xman »
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #34 on: June 17, 2010, 02:09:42 PM »
What do you think?

I think you haven't a clue.
Beware Romanians bearing strange gifts

Offline veritas

  • Member
  • Posts: 1,408
Re: I ♥ Gene Therapy
« Reply #35 on: June 17, 2010, 02:34:00 PM »

Inch, Free,

Nice finds as usual ! Let's hit this thing any way we can. It will Happen!

Mercury,

Keep up the good fight ! I'll second what Inch said: Your definitely an inspiration!
We're all rooting for you!! Your the very definition of: " It is better to light one small candle, than to curse the darkness".

v

Offline tennisplayer56

  • Member
  • Posts: 25
Re: I ♥ Gene Therapy
« Reply #36 on: July 04, 2010, 06:54:54 PM »
If you read very carefully the second article you'll notice that a transplant and a shutdown of the bown marrow and immune system was made as for the german patient in Berlin. They also state that they're studying less toxic ways to follow such a procedure and to have the same outcome. Less toxic can meaning everything. This it what concerns me more. It could be still lifethreatening!

Dramaticcccc. =). Its all about relativity. Aspirin is potentially "life-threatening", as it can cause ulcers, tinnitis, etc. Obviously the FDA is not going to pass something that won't be similar or better than current therapies, AND be non-lifethreatening. I'm sure science, in its own time, will find ways to replicate BMT's with less toxicity.

Offline geobee

  • Member
  • Posts: 261
Re: I ♥ Gene Therapy
« Reply #37 on: July 30, 2010, 11:24:45 AM »
Here's a radio program with Paula Cannon, others, discussing gene therapies.  There are some commercials in it -- but it's still worthwhile.  I had to click on the download link to hear it.

http://www.scpr.org/programs/airtalk/2010/07/21/stem-cell-research-aimed-at-curing-aids/

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #38 on: July 30, 2010, 12:27:46 PM »
Here's a radio program with Paula Cannon, others, discussing gene therapies.  There are some commercials in it -- but it's still worthwhile.  I had to click on the download link to hear it.

http://www.scpr.org/programs/airtalk/2010/07/21/stem-cell-research-aimed-at-curing-aids/

Thanks for that link, it was very interesting.

I love Paula Cannon's accent, I guess she's Australian?
« Last Edit: July 30, 2010, 12:38:12 PM by Inchlingblue »

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #39 on: July 30, 2010, 01:48:17 PM »
.
I love Paula Cannon's accent, I guess she's Australian?

I'm going to vote for her being English.  Undergrad and PhD from University of Liverpool and Post Doctoral Work at Oxford.  Besides, she doesn't seem to have that upward inflection at the end of many of her words our mates from Oz seem to have :)

Dr. Cannon ♥ Gene Therapy too!
Beware Romanians bearing strange gifts

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #40 on: July 30, 2010, 02:45:22 PM »
Not specifically about HIV, interesting nevertheless...

http://health.usnews.com/health-news/family-health/cancer/articles/2010/07/21/gene-therapy-shows-promise-with-bubble-boy-disease.html

Gene Therapy Shows Promise With 'Bubble Boy' Disease

WEDNESDAY, July 21 (HealthDay News) -- Eight of nine male infants born with so-called "Bubble Boy" disease were still alive and well nine years after they underwent gene therapy, French researchers report.


Up to 11 years after the therapy, most patients had normal T-cell levels and were able to lead normal lives, including attending regular schools. Their weight and height were not stunted, as is usually the case with this condition.

But the gains did not come without a price. Almost half of the participants in the study developed acute leukemia after the therapy. Three survived, while one died.

Still, said William J. Bowers, an associate professor of neurology at the University of Rochester Medical Center in New York, "this is a very substantial advance."

And, he added, since these procedures were performed (between 1999 and 2002), additional advances have been made in terms of the virus vectors used to insert the corrected genes which, once tested in patients, could reduce the risk of cancer.

The virus vector used in this trial inadvertently activated an oncogene, which led to the development of the leukemia.

"Bubble Boy" disease, known as X-linked severe combined immunodeficiency (SCID-X1), occurs in at least one in every 50,000 to 100,000 newborns, according to the National Institutes of Health.

It's caused by mutations in the IL2RG gene, which interferes with the normal production of immune system cells known as lymphocytes.

Boys with the condition -- only boys inherit the gene -- have no T-cells and so are unable to fight off infections.

The first treatment of choice for these patients is a bone marrow or cord blood transplant, but that requires a matched donor and can involve serious complications.

The nine boys in this study -- who had a median age of 7 months at the time they received the corrected gene -- were not eligible for transplants because no good matches were available.

Researchers removed CD34+ cells, a type of stem cell found in the bone marrow, added a corrected gene, then reinserted them into the respective patients, all of whom were placed in isolation.

The patients were able to leave the isolation unit 45 to 90 days after the procedure, and none developed any severe infections, although all did get Varicella zoster virus (which causes chicken pox). The infection wasn't severe enough to require hospitalization.

Some of the patients also had chronic rhinitis (runny nose) and two patients developed warts, though these subsequently went away.

The most serious side effect was the development of T-cell acute lymphoblastic leukemia in three patients, one of whom died. The other three successfully underwent chemotherapy.

Participants all had healthy T-cells up to 11 years after the initial therapy.

"I think the field of gene therapy is starting to see clinical successes and is going to go forward fairly rapidly," Bowers said.

The study appears in the July 22 issue of the New England Journal of Medicine.
Beware Romanians bearing strange gifts

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #41 on: July 30, 2010, 03:33:32 PM »
When a Nobel winning scientist like Dr. Baltimore says that a 10 year estimate for one of the "exciting" stem cell therapies to come on line is an "overstatement", i.e. will happen sooner, I sit up and take notice.

One other note about the interview.  Love how Dr. Baltimore shuts down an asshole who calls in; some guy "Jack", with his lame "too much funding for a preventable disease vs. unpreventable" meme.  No doubt Jack is a Sarah Palin supporter.
« Last Edit: July 30, 2010, 08:19:58 PM by freewillie99 »
Beware Romanians bearing strange gifts

Offline Inchlingblue

  • Member
  • Posts: 3,119
  • Chad Ochocinco PETA Ad
Re: I ♥ Gene Therapy
« Reply #42 on: November 14, 2010, 08:24:33 AM »
This article gives a good overview of upcoming gene therapy trials.

Clinical Trials Explore Gene Therapy For The Treatment Of HIV


LINK:

http://www.aidsbeacon.com/news/2010/11/12/clinical-trials-explore-gene-therapy-for-the-treatment-of-hiv-aids/

Offline ElZorro

  • Member
  • Posts: 535
Re: I ♥ Gene Therapy
« Reply #43 on: November 14, 2010, 10:11:37 AM »
Interesting stuff. A tad disappointed that the City of Hope trial will take 15 years, but all good things are worth waiting for I guess

Offline georgep77

  • Member
  • Posts: 145
Re: I ♥ Gene Therapy
« Reply #44 on: November 14, 2010, 10:05:53 PM »
Interesting stuff. A tad disappointed that the City of Hope trial will take 15 years

15 years !!..... with all respect, that's kinda LOL

              ;D
« Last Edit: November 14, 2010, 10:07:49 PM by georgep77 »
Come on Sangamo,  Geovax,  Bionor immuno, ...Make us happy !!!
+ 2008

Granny60

  • Guest
Re: I ♥ Gene Therapy
« Reply #45 on: November 14, 2010, 10:15:02 PM »
Yep, I have friends that like Gene therapy,  also Michael,  and Steven and Tom and Susan...... ;D

Offline freewillie99

  • Member
  • Posts: 304
Re: I ♥ Gene Therapy
« Reply #46 on: November 15, 2010, 02:02:42 PM »
Yep, I have friends that like Gene therapy,  also Michael,  and Steven and Tom and Susan...... ;D

moronic.
Beware Romanians bearing strange gifts

Offline sensual1973

  • Member
  • Posts: 195
Re: I ♥ Gene Therapy
« Reply #47 on: November 15, 2010, 05:56:01 PM »
15 years ? hope i will still be alive !
« Last Edit: November 15, 2010, 06:12:05 PM by sensual1973 »
God grant me the serenity to accept the things i can not change.

Offline xman

  • Member
  • Posts: 283
Re: I ♥ Gene Therapy
« Reply #48 on: November 15, 2010, 06:44:08 PM »
i guess the 15 years time frame is to evaluate delayed adverse effects. they will see much earlier if the treatment is working or not.
sign the petition launched by the aids policy project addressed to the nih aimed to increase the money needed to find the cure:

http://www.aidspolicyproject.org/petition_for_the_nih

we can make a difference and we need to fight. please support them! it doesn't cost you anything. they need it now more than ever!

Offline ElZorro

  • Member
  • Posts: 535
Re: I ♥ Gene Therapy
« Reply #49 on: November 16, 2010, 08:48:15 AM »
i guess the 15 years time frame is to evaluate delayed adverse effects. they will see much earlier if the treatment is working or not.

I'm sure you're right XMan. And, considering they are actually messing with genes, this could result in creating a true set of mutant XMen  ;)

 


Terms of Membership for these forums
 

© 2014 Smart + Strong. All Rights Reserved.   terms of use and your privacy
Smart + Strong® is a registered trademark of CDM Publishing, LLC.