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 infection and 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. According to the CDC, more than 1.1 million people in the U.S. are infected with HIV at this moment.

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.

Clinical Stage Programs

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 team has begun a clinical trial with the procedure.


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. The team has completed a Phase 1 clinical trial with the procedure.

University of California, Davis

The team at is taking a patient’s blood forming stem cells and inserting 3 anti-HIV genes into them and then returning them to the individual. The anti-HIV genes are then passed on to all new immune system cells and make them resistant to HIV. Because AIDS-related lymphoma is linked to the constant immune cell stimulation caused by HIV infection, getting rid of the virus should prevent return of the cancer.

University of California, San Francisco

The team will take a patient’s blood and extract T cells, a type of immune cell.  The T cells are then genetically modified to express two different chimeric antigen receptors (CAR), which enable the newly created duoCAR-T cells to recognize and destroy HIV infected cells.  The modified T cells are then reintroduced back into the patient. The goal of this one-time therapy is to act as a long-term control of HIV with patients no longer needing to take ART, in effect a form of HIV cure. 

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 NameInstitutionGrant TitleGrant TypeAward Amount
Brian LawsonThe Scintillon InstituteAn hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies.Quest – Discovery Stage Research Projects$1,143,600
Dr. Scott G KitchenUniversity of California, Los AngelesEngineering Lifelong Cellular Immunity to HIVQuest – Discovery Stage Research Projects$1,701,178
Dr. David Baltimore PhDCalifornia Institute of TechnologyImmunotherapy for HIV infection using engineered hematopoietic stem/progenitor cellsQuest – Discovery Stage Research Projects$1,569,193
Dr. Scott G KitchenUniversity of California, Los AngelesHematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV InfectionTherapeutic Translational Research Projects$6,140,723
Dr. Angelo Manuel Almeida Cardoso Ph.D., MDCity of Hope, Beckman Research InstituteEvaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIVClinical Trial Stage Projects$11,299,976
William KennedyExcision BioTherapeuticsPhase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapyClinical Trial Stage Projects$6,852,486
Steven G. DeeksUniversity of California, San FranciscoAnti-HIV duoCAR-T cell therapy for HIV infectionClinical Trial Stage Projects$8,970,732
Dr Mehrdad AbediUniversity of California, DavisStem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patientsClinical Trial Stage Projects$8,414,265
Xiuli WangCity of Hope, Beckman Research InstituteCMV-specific T cells expressing anti-HIV CAR and CMV vaccine boost as immunotherapy for HIV/AIDSLate Stage Preclinical Projects$3,812,797
Dr. Paula M. CannonUniversity of Southern CaliforniaSite-specific gene editing in hematopoietic stem cells as an anti-HIV therapyTools and Technologies III$1,495,665
Dr. Irvin SY ChenUniversity of California, Los AngelesStem Cells: A New Avenue of HIV Research and New Approaches to HIV TreatmentConference$24,456
Prof. John A ZaiaCity of Hope, Beckman Research InstituteA 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 (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Sub…Strategic Partnership III Track A$5,583,438
Dr. Mark S. AndersonUniversity of California, San FranciscoGeneration of a functional thymus to induce immune tolerance to stem cell derivativesBasic Biology V$1,191,000
Dr. Irvin SY ChenUniversity of California, Los AngelesDevelopment of a humanized mouse model for testing anti-HIV HSPC gene therapy strategies in HIV-1 infected mice.Early Translational from Disease Team Conversion$1,505,000
Dr. Jerome A. Zack Ph.D.University of California, Los AngelesStem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV InfectionEarly Translational IV$4,925,166
Dr Mehrdad AbediUniversity of California, DavisStem Cell Gene Therapy for HIV in AIDS Lymphoma PatientsDisease Team Therapy Planning I$66,880
Dr. David Louis DiGiustoCity of Hope, Beckman Research InstituteDevelopment of RNA-based approaches to stem cell gene therapy for HIVEarly Translational II$3,097,160
Dr. Mark S. AndersonUniversity of California, San FranciscoStem cell differentiation to thymic epithelium for inducing tolerance to stem cellsTransplantation Immunology$1,314,089
Dr. Jerome A. Zack Ph.D.University of California, Los AngelesHuman Embryonic Stem Cell Therapeutic Strategies to Target HIV DiseaseComprehensive Grant$2,401,903
Dr. Irvin SY ChenUniversity of California, Los AngelesGenetic modification of the human genome to resist HIV-1 infection and/or disease progressionSEED Grant$616,800
Dr. Irvin SY ChenUniversity of California, Los AngelesHPSC based therapy for HIV disease using RNAi to CCR5.Disease Team Research I$9,905,604
Prof. John A ZaiaCity of Hope, Beckman Research InstituteZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDSDisease Team Research I$14,536,969


Is a HIV Cure Possible? A Lecture Series on HIV Cure Research

Is a HIV Cure Possible? A Panel Discussion on HIV Cure Research

HIV/AIDS Stem Cell Clinical Trial: Introduction by Jeff Sheehy

HIV/AIDS Stem Cell Clinical Trial: Louis Breton, CEO, Calimmune

HIV/AIDS Stem Cell Clinical Trial: Geoff Symonds, Calimmune

John Zaia, City of Hope – CIRM Stem Cell #SciencePitch

HIV/AIDS: Advancing Stem Cell Therapies: 2011 CIRM Grantee Meeting

Progress and Promise in HIV/AIDS

Spotlight on Disease Team Awards – HIV/AIDS: Jeff Sheehy

Spotlight on Disease Team Awards – HIV/AIDS: John Zaia

Spotlight on Disease Team Awards – HIV/AIDS: Loring Leeds

Biotech Perspectives for Stem Cell Research: Don Francis

HIV and Stem Cells: Warner Greene – CIRM Science Writer’s Seminar


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