HIV/AIDS Fact Sheet

CIRM funds many projects seeking to better understand HIV/AIDS and to translate those discoveries into new therapies.


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.


The Calimmune 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.

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 Approved funds
David DiGiusto City of Hope Development of RNA-based approaches to stem cell gene therapy for HIV $3,097,160
John Zaia City of Hope ZIinc Finger Nuclease-Based Stem Cell Therapy for AIDS $14,536,969
Irvin Chen University of California, Los Angeles HPSC based therapy for HIV disease using RNAi to CCR5. $9,905,604
Irvin Chen University of California, Los Angeles Genetic modification of the human genome to resist HIV-1 infection and/or disease progression $616,800
Zack Jerome University of California, Los Angeles Human Embryonic Stem Cell Therapeutic Strategies to Target HIV Disease $2,401,903
Mark Anderson University of California, San Francisco Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells $1,314,089
Mehrdad Abedi University of California, Davis RUNNING TITLE: Stem Cell Gene Therapy for HIV in AIDS Lymphoma Patients $66,880
Zack Jerome University of California, Los Angeles Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection $4,925,166
Irvin Chen University of California, Los Angeles HPSC based therapy for HIV disease using RNAi to CCR5. $1,505,000
Mark Anderson University of California, San Francisco Generation of a functional thymus to induce immune tolerance to stem cell derivatives $1,191,000
John Zaia City of Hope A Phase I, Open-Label Study to Assess the Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells with Escalating Doses of Busulfan in HIV-1 (R5) Infected Subjects with Suboptim $5,583,438
John Zaia City of Hope The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases. $8,000,000
Paula Cannon University of Southern California Site-specific gene editing in hematopoietic stem cells as an anti-HIV therapy $1,499,400
Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients $8,414,265


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