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Gene therapy for hemophilia: what does the future hold?

Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in hemophilia A and B. These trials, which highlight the challenges overcome through decades of preclinical and first in human clinical studies, ha...

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Dades bibliogràfiques
Publicat a:Ther Adv Hematol
Autors principals: Doshi, Bhavya S., Arruda, Valder R.
Format: Artigo
Idioma:Inglês
Publicat: SAGE Publications 2018
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6130099/
https://ncbi.nlm.nih.gov/pubmed/30210756
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/2040620718791933
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