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Update on clinical gene therapy for hemophilia

In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are currently in clinical development, gene therapy holds the promise of a lasting cure with a single drug administration. Near-to-complete correction of hemophilia A (factor VIII deficiency) and hemophilia B (...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Blood
Prif Awduron: Perrin, George Q., Herzog, Roland W., Markusic, David M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Hematology 2019
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC6356985/
https://ncbi.nlm.nih.gov/pubmed/30559260
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2018-07-820720
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