Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

Recently, we demonstrated that hematopoietic stem/progenitor cell (HSPC) mobilization followed by intravenous injection of integrating, helper-dependent adenovirus HDAd5/35++ vectors resulted in efficient transduction of long-term repopulating cells and disease amelioration in mouse models after in...

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Dettagli Bibliografici
Pubblicato in:JCI Insight
Autori principali: Wang, Hongjie, Georgakopoulou, Aphrodite, Li, Chang, Liu, Zhinan, Gil, Sucheol, Bashyam, Ashvin, Yannaki, Evangelia, Anagnostopoulos, Achilles, Pande, Amit, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Lieber, André
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2020
Soggetti:
Technical Advance
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7455141/
https://ncbi.nlm.nih.gov/pubmed/32814708
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.139538
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