Current Progress in Therapeutic Gene Editing for Monogenic Diseases
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference117 articles.
1. Efficacy of gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N Engl J Med,2010
2. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union;Ylä-Herttuala;Mol Ther,2012
3. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial;Alton;Lancet Respir Med,2015
4. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering;Gaj;Trends Biotechnol,2013
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