CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

A young man with HIV infection and acute lymphoblastic leukemia, both in remission, underwent bone marrow transplantation with donor stem cells, a minority of which had undergone disruption of the CCR5 gene. The manipulated cells engrafted. Only a small percentage of the CD4+ cells carried the disru...

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Veröffentlicht in:	The New England journal of medicine 2019-09, Vol.381 (13), p.1240-1247
Hauptverfasser:	Xu, Lei, Wang, Jun, Liu, Yulin, Xie, Liangfu, Su, Bin, Mou, Danlei, Wang, Longteng, Liu, Tingting, Wang, Xiaobao, Zhang, Bin, Zhao, Long, Hu, Liangding, Ning, Hongmei, Zhang, Yufeng, Deng, Kai, Liu, Lifeng, Lu, Xiaofan, Zhang, Tong, Xu, Jun, Li, Cheng, Wu, Hao, Deng, Hongkui, Chen, Hu
Format:	Artikel
Sprache:	eng
Schlagworte:	Ablation Acute lymphoblastic leukemia Adult Anti-Retroviral Agents - therapeutic use Antiretroviral drugs Blood Blood Cell Count Blood cells Bone marrow CCR5 protein CD4 antigen CD4 Lymphocyte Count Chemotherapy Chimerism CRISPR CRISPR-Cas Systems Drug dosages Efficiency Gene Editing - methods Gene therapy Genome editing Genomes Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells HIV HIV Infections - complications HIV Infections - drug therapy HIV Infections - therapy HIV-1 - genetics Human immunodeficiency virus Humans Infections Laboratories Leukemia Life sciences Lymphatic leukemia Lymphocytes Male Mutation Precursor Cell Lymphoblastic Leukemia-Lymphoma - complications Progenitor cells R&D Receptors, CCR5 - genetics Remission Research & development Transplantation Viral Load
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Beschreibung
Zusammenfassung:	A young man with HIV infection and acute lymphoblastic leukemia, both in remission, underwent bone marrow transplantation with donor stem cells, a minority of which had undergone disruption of the CCR5 gene. The manipulated cells engrafted. Only a small percentage of the CD4+ cells carried the disrupted gene, but such cells survived at least 19 months.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1817426