Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release-Reference-Cited by-同舟云学术

Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

Published:2020-06 Issue: Volume:17 Page:581-588
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Lengler Johannes,Coulibaly Sogue,Gruber Bernadette,Ilk Reinhard,Mayrhofer Josef,Scheiflinger Friedrich,Hoellriegl Werner,Falkner Falko G.,Rottensteiner Hanspeter

Funder

Baxalta Innovations GmbH

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

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4. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N. Engl. J. Med.,2011

5. X-linked thrombophilia with a mutant factor IX (factor IX Padua);Simioni;N. Engl. J. Med.,2009

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