Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Lentivector gene therapy for X-linked chronic granulomatous disease (X-CGD) has proven to be a viable approach, but random vector integration and subnormal protein production from exogenous promoters in transduced cells remain concerning for long-term safety and efficacy. A previous genome editing–b...

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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Blood
Egile Nagusiak: De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Liu, Siyuan, Li, Linhong, Pavel-Dinu, Mara, Lazzarotto, Cicera R., Liu, Taylor, Koontz, Sherry M., Choi, Uimook, Sweeney, Colin L., Theobald, Narda, Lee, GaHyun, Clark, Aaron B., Burkett, Sandra S., Kleinstiver, Benjamin P., Porteus, Matthew H., Tsai, Shengdar, Kuhns, Douglas B., Dahl, Gary A., Headey, Stephen, Wu, Xiaolin, Malech, Harry L.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Hematology 2021
Gaiak:
Gene Therapy
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC8120141/
https://ncbi.nlm.nih.gov/pubmed/33623984
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood.2020008503
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