HRA002123
Title:
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
Release date:
2023-12-14
Description:
We transplanted CRISPR-edited CCR5-ablated hematopoietic stem and progenitor cells (HSPCs) into a patient with HIV-1 infection and acute lymphoblastic leukemia. The acute lymphoblastic leukemia was in complete remission with full donor chimerism, and donor cells carrying the ablated CCR5 persisted for more than 19 months without gene editing-related adverse events. The percentage of CD4+ cells with CCR5 ablation increased by a small degree during a period of antiretroviral-therapy interruption. Although we achieved successful transplantation and long-term engraftment of CRISPR-edited HSPCs, the percentage of CCR5 disruption in lymphocytes was only approximately 5%, which indicates the need for further research into this approach.
Data Accessibility:   
Open access
BioProject:
Study type:
Disease Study
Disease name:
human immunodeficiency virus infectious disease
Individuals & samples
Files
Submitter:   Deng Hongkui / hongkui_deng@pku.edu.cn
Organization:   Peking University
Submission date:   2022-03-15