HIV/AIDS Fact Sheet

CIRM funds several research projects investigating ways of modifying adult blood-forming stem cells or embryonic stem cells to create a replacement immune system that is resistant to HIV infection.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

Stem cell research for HIV/AIDS

HIV, or the human immunodeficiency virus, is a virus that infects cells of the immune system, undermining the body’s ability to fight disease. Eventually infection can lead to symptoms of AIDS (Acquired Immune Deficiency Syndrome), which includes susceptibility to infections, cancers and other diseases, and eventually causes death. The CDC estimated that in 2010, more than 1.148 million people in the U.S. were infected with HIV.

Stem cell approaches to treating people with HIV primarily involve replacing the person’s immune system with one that the virus can’t infect. Hope that this approach could work were boosted in late 2010 when scientists reported that Timothy Ray Brown, also known as the "Berlin Patient”, had effectively had his HIV “cured”. As part of a treatment for leukemia, Brown had received a bone marrow transplant that came from a donor whose cells were resistant to HIV infection.

The person who donated the bone marrow had a genetic mutation in a gene called CCR5, which makes a protein that is required for HIV to enter cells. Without CCR5, HIV wasn’t able to infect these replacement immune cells and Brown has been able to go off his medications.

The problem is that there aren’t enough people with naturally occurring CCR5 mutations to serve as bone marrow donors for all HIV patients. Instead, scientists are hoping to create CCR5 mutations. They first plan to remove the blood-forming stem cells in a person’s bone marrow and mutate the CCR5 gene. The idea is that those genetically altered cells would then repopulate the person’s blood system with one that lacks CCR5 and that HIV won’t be able to infect.

Disease Teams

City of Hope

The City of Hope team plans to mutate the CCR5 gene using a technology called a zinc finger nuclease, which is essentially a pair of molecular scissors developed by Sangamo Biosciences that snips an exact spot on the CCR5 gene. Early evidence in animals suggests that when those cells are reintroduced, they create an immune system that HIV can’t infect.

Read a summary of this project

University of California, Los Angeles

The UCLA team is using a method called RNA interference to block the CCR5 gene from generating a protein. A blood system generated from these cells will lack CCR5 and block HIV infection.

Read a summary of this project

Jeff Sheehy, HIV/AIDS patient advocate member of the CIRM Governing Board, and John Zaia, leader of the City of Hope CIRM HIV Disease Team, discuss stem cell transplant strategies for the treatment of HIV/AIDS.

CIRM Grants Targeting HIV/AIDS

Researcher name	Institution	Grant Title	Funding
John Zaia	Beckman Research Institute of City of Hope	ZIinc Finger Nuclease-Based Stem Cell Therapy for AIDS	$14,583,187
Irvin Chen	University of California, Los Angeles	HPSC based therapy for HIV disease using RNAi to CCR5.	$9,905,604
Geoff Symonds	Calimmune, Inc.	GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE	$8,278,722
Zack Jerome	University of California, Los Angeles	Human Embryonic Stem Cell Therapeutic Strategies to Target HIV Disease	$2,516,831
Irvin Chen	University of California, Los Angeles	Genetic modification of the human genome to resist HIV-1 infection and/or disease progression	$642,652
David DiGiusto	Beckman Research Institute of City of Hope	Development of RNA-based approaches to stem cell gene therapy for HIV	$3,124,130
			Total: $39,051,126.00