Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques

Adeno-associated viral vectors (AAVs) achieve stable therapeutic expression without long-term toxicity in adults with hemophilia. To avert irreversible complications in congenital disorders producing early pathogenesis, safety and efficacy of AAV-intrauterine gene transfer (IUGT) requires assessment...

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Detalhes bibliográficos
Publicado no:FASEB J
Main Authors: Chan, Jerry K. Y., Gil-Farina, Irene, Johana, Nuryanti, Rosales, Cecilia, Tan, Yi Wan, Ceiler, Jessika, Mcintosh, Jenny, Ogden, Bryan, Waddington, Simon N., Schmidt, Manfred, Biswas, Arijit, Choolani, Mahesh, Nathwani, Amit C., Mattar, Citra N. Z.
Formato: Artigo
Idioma:Inglês
Publicado em: Federation of American Societies for Experimental Biology 2019
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6404563/
https://ncbi.nlm.nih.gov/pubmed/30517034
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1096/fj.201801391R
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