Gene Therapy for Hemophilia

The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [FVIII] for hemophilia A and factor IX [FIX] for hemophilia B). H...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther
Prif Awduron: Nienhuis, Arthur W., Nathwani, Amit C., Davidoff, Andrew M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2017
Pynciau:
Review
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC5417837/
https://ncbi.nlm.nih.gov/pubmed/28411016
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.03.033
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