CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
| Title | CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia |
|---|---|
| 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. |
| Organism | Homo sapiens |
| Data Type | Genotype |
| Data Accessibility | Open-access |
| BioProject | PRJCA008651 |
| Release Date | 2023-12-16 |
| Submitter | Hongkui Deng (hongkui_deng@pku.edu.cn) |
| Organization | Peking University |
| Submission Date | 2022-03-15 |
The data cannot be downloaded as it has not yet been registered in the Human Genetic Resource Management Platform of MOST.
| File ID | File Title | Number/Samples | File Type | File Size | File Suffix | Download Times | Download |
|---|---|---|---|---|---|---|---|
| OMIX001035-06-01 | sp15 gene mutation analysis.xls | 1 | Genotype | 46.6 KB | xls | 0 | Unavailable |
| OMIX001035-06-02 | sp44 gene mutation analysis.xls | 1 | Genotype | 55.4 KB | xls | 0 | Unavailable |