In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

Current thalassemia gene therapy protocols require the collection of hematopoietic stem/progenitor cells (HSPCs), in vitro culture, lentivirus vector transduction, and retransplantation into myeloablated patients. Because of cost and technical complexity, it is unlikely that such protocols will be a...

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Veröffentlicht in:J Clin Invest
Hauptverfasser: Wang, Hongjie, Georgakopoulou, Aphrodite, Psatha, Nikoletta, Li, Chang, Capsali, Chrysi, Samal, Himanshu Bhusan, Anagnostopoulos, Achilles, Ehrhardt, Anja, Izsvák, Zsuzsanna, Papayannopoulou, Thalia, Yannaki, Evangelia, Lieber, André
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2018
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6355219/
https://ncbi.nlm.nih.gov/pubmed/30422819
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI122836
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