Page URL:

Potential breakthrough in HIV gene therapy

7 March 2011
Appeared in BioNews 598

American researchers have successfully created immune cells resistant to HIV (human immunodeficiency virus). T cells, which are the main target of HIV, were isolated from six HIV positive patients and genetically manipulated to confer resistance. The cells were injected back into the same patients and were able to survive and multiply. The results of the phase I clinical trial do not provide a cure, but suggest gene therapy may be a viable treatment in the future.

'This is the first successful example of targeted genetic modification of the DNA code in patients, and therefore this has implications for the development of corrective gene therapy for a number of monogenic gene disorders of the bone marrow, such as sickle cell anemia, that are currently incurable', said Dr Carl June, lead researcher and director of translational research at the Abramson Cancer Centre at the University of Pennsylvania.

The trial was based on the case of the 'Berlin patient', a patient with AIDS (acquired immunodeficiency syndrome) living in Germany who received a blood transfusion from an individual with natural resistance to HIV. The patient was 'functionally cured', as the virus could no longer be detected even after four years. Natural resistance to HIV, such as that of the blood donor in the case of the 'Berlin patient', occurs in a small percentage of people who lack the CCR5 gene. CCR5 is a cell surface receptor present on T cells, which HIV commandeers to infect the cells.

Researchers at the biotechnology company, Sangamo BioSciences in California, genetically manipulated T cells of HIV infected patients, using zinc finger technology. The CCR5 gene was permanently removed from the T cells, before reintroducing the cells back into the bloodstream of the patients. The cells were able to survive for at least three months, as well as increase in number.

'The procedure is safe so far and feasible, in that all patients can be treated i.e. no manufacturing failures, and thus the phase I protocol has met its objectives and can be deemed 'successful'', said Dr June.

Experts in the field point out that the results are far from a cure for HIV. 'If successful, this probably could have wide application, but going from six patients to an entire epidemic is a ways to go', said Dr Michael Horberg, vice chair of the HIV Medicine Association.

The results of the trial were presented at the 18th Conference on Retroviruses and Opportunistic Infections in Boston, which took place from 27 February to 2 March 2011.

Gene Therapy Against HIV Not a Proven Cure, Experts Say
Business Week |  03/11
Gene therapy raises hope for a future AIDS cure
Press Association |  03/11
Giving HIV a Poor Reception: New AIDS Treatment Tinkers with Immune Cell Genes
Scientific American |  3 March 2011
Promising new gene therapy for HIV immunity
Philadelphia Inquirer |  3 March 2011
Scientists tweak cells so they can defy HIV
Sydney Morning Herald |  4 March 2011
Targeted gene editing enters clinic
Nature |  03/11
20 November 2017 - by Jen Willows 
A new clinical trial in California marks the first time that genome editing has been used inside the body, rather than on cells such as blood or skin which can be extracted, edited outside the body, and then replaced...
19 October 2015 - by Darius Khalid 
This book is an impressive examination of the CCR5 gene, which provides a carefully detailed interdisciplinary understanding involving biochemistry, genetics and biotechnology, as well as the historical and contemporary aspects of patenting and intellectual property laws...
8 May 2012 - by Helen Brooks 
HIV patients treated over a decade ago with genetically modified immune cells have suffered no serious side effects from the treatment. Although the cells may not have been successful in targeting and killing HIV-infected cells, the results are promising as they bolster the safety credentials of gene therapy for clinical use...
5 December 2011 - by Dr Tamara Hirsch 
US scientists have induced long-lasting HIV protection in mice from a single injection. Their study, published in the journal Nature, uses gene therapy to stimulate production of antibodies against the virus...
26 September 2011 - by Dr Louisa Petchey 
The success of a new gene therapy trial represents a significant step towards a 'functional cure' for HIV, US researchers announced this week. By mimicking the effects of a naturally occurring gene mutation that makes an individual resistant to infection, this therapy aims to reduce or eliminate the dependency of HIV patients on antiretroviral drugs....
27 September 2010 - by Dr Jay Stone 
Questions continue to be asked after Dr Savio Woo, a gene therapist at the Mount Sinai School of Medicine in New York, was forced to retract two more of his papers last week. Dr Woo has retracted six papers this year after two of his post-docs, Li Chen and Zhiyu Li, were accused of scientific misconduct....
20 September 2010 - by Dr Lux Fatimathas 
A patient with the genetic blood disorder beta-thalassaemia has been successfully treated using genetically-altered stem cells, according to US and French researchers...
21 June 2010 - by Dr Lux Fatimathas 
A human RNA-based gene therapy trial to combat HIV has passed the first safety test. US researchers modified human blood stem cells to make them resistant to the virus....
8 March 2010 - by Ruth Pidsley 
A team of researchers based at Ohio State University, USA, have used gene therapy to restore nerve and muscle function and prolong life in mice with a form of spinal muscular atrophy (SMA), a lethal childhood muscle-wasting disorder. Results from the study, published in the journal Nature Biotechnology, were so encouraging that the researchers hope to progress to human trials within two years...
15 November 2009 - by Alison Cranage 
Research published in the journal Science Translational Medicine last week shows gene therapy can improve muscle size and strength in monkeys. The technique holds promise as a therapy for several neuromuscular disorders, and researchers hope that clinical trials will start next year....
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.