Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells
Author:
Funder
BioMarin Pharmaceutical
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference86 articles.
1. Nucleotide sequence and organization of the adeno-associated virus 2 genome;Srivastava;J. Virol.,1983
2. AAV-mediated gene therapy for research and therapeutic purposes;Samulski;Annu. Rev. Virol.,2014
3. Adeno-associated virus vector as a platform for gene therapy delivery;Wang;Nat. Rev. Drug Discov.,2019
4. A five-repeat micro-dystrophin gene ameliorated dystrophic phenotype in the severe DBA/2J-mdx model of Duchenne muscular dystrophy;Hakim;Mol. Ther. Methods Clin. Dev.,2017
5. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy;Harper;Nat. Med.,2002
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