| Accession |
PRJCA008651 |
| Title |
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia |
| Relevance |
Medical |
| Data types |
Whole genome sequencing
Raw sequence reads
Genome sequencing
|
| Organisms |
Homo sapiens
|
| Description |
The safety of CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing in the context of human gene therapy is largely unknown. CCR5 is a reasonable but not absolutely protective target for a cure of human immunodeficiency virus type 1 (HIV-1) infection, because CCR5-null blood cells are largely resistant to HIV-1 entry. 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. |
| Sample scope |
Multiisolate |
| Release date |
2023-12-14 |
| Publication |
| PubMed ID |
Article title |
Journal name |
DOI |
Year |
| 31509667
|
|
|
|
|
|
|
| Grants |
| Agency |
program |
Grant ID |
Grant title |
| Ministry of Science and Technology of the People's Republic of China (MOST)
|
|
2017YFA0103000
|
|
|
|
| Submitter |
Hongkui
Deng (hongkui_deng@pku.edu.cn)
|
| Organization |
Peking University |
| Submission date |
2022-03-14 |
