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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Pubblicato in:Blood
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2021
Soggetti:
Gene Therapy
Accesso online: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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