CRISPR-Edited CCR5-Ablated Stem Cells Persist in a Person with HIV and Leukemia

HIV uses the coreceptor CCR5 to infect CD4+ T cells. Two people have been cured of HIV after being treated for malignancy with stem cell transplants from donors who lack this coreceptor. Now, investigators in China have used CRISPR (clustered regularly interspaced short palindromic repeats)–Cas9, a promising gene-editing tool, to ablate CCR5 from donor cells given to a person with HIV and acute lymphocytic leukemia (ALL).

The recipient had CCR5-tropic HIV infection and was receiving antiretroviral therapy (ART), resulting in undetectable plasma HIV RNA. He had been diagnosed with ALL and was in remission after standard chemotherapy. He then underwent an allogeneic stem cell transplant from an HLA-matched donor who had wild-type CCR5. The rec…