Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins-Reference-Cited by-同舟云学术

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Published:2011-05 Issue:5 Volume:19 Page:876-885
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Nathwani Amit C,Rosales Cecilia,McIntosh Jenny,Rastegarlari Ghasem,Nathwani Devhrut,Raj Deepak,Nawathe Sushmita,Waddington Simon N,Bronson Roderick,Jackson Scott,Donahue Robert E,High Katherine A,Mingozzi Federico,Ng Catherine YC,Zhou Junfang,Spence Yunyu,McCarville M Beth,Valentine Marc,Allay James,Coleman John,Sleep Susan,Gray John T,Nienhuis Arthur W,Davidoff Andrew M

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference46 articles.

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4. A factor IX-deficient mouse model for hemophilia B gene therapy;Wang;Proc Natl Acad Sci USA,1997

5. Prospects for gene therapy of haemophilia;Nathwani;Haemophilia,2004

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