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Defenestrated endothelium delays liver-directed gene transfer in hemophilia A mice

Hemophilia A is an inherited bleeding disorder caused by defective or deficient coagulation factor VIII (FVIII) activity. Until recently, the only treatment for prevention of bleeding involved IV administration of FVIII. Gene therapy with adeno-associated vectors (AAVs) has shown some efficacy in pa...

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Detalles Bibliográficos
Autores principales:	Kaminski, Tomasz W., Ju, Eun-Mi, Gudapati, Shweta, Vats, Ravi, Arshad, Sanya, Dubey, Rikesh K., Katoch, Omika, Tutuncuoglu, Egemen, Frank, Jonathan, Brzoska, Tomasz, Stolz, Donna B., Watkins, Simon C., Chan, Stephen Y., Ragni, Margaret V., Novelli, Enrico M., Sundd, Prithu, Pradhan-Sundd, Tirthadipa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Hematology 2022
Materias:	Stimulus Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9631574/ https://www.ncbi.nlm.nih.gov/pubmed/35427414 http://dx.doi.org/10.1182/bloodadvances.2021006388

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