AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer-Reference-Cited by-同舟云学术

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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Published:2020-03 Issue:3 Volume:28 Page:723-746
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Verdera Helena Costa,Kuranda Klaudia,Mingozzi Federico

Funder

European Research Council

Publisher

Elsevier BV

Subject

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

Reference236 articles.

1. Terminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteins;Lovric;Mol. Ther.,2012

2. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial;Bennett;Lancet,2016

3. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer;Buchlis;Blood,2012

4. 5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency;Mueller;Mol. Ther.,2017

5. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N. Engl. J. Med.,2014

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