Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers
Author:
Publisher
Elsevier BV
Subject
General Biochemistry, Genetics and Molecular Biology
Reference61 articles.
1. Duchenne muscular dystrophy;Duan;Nat. Rev. Dis. Primers,2021
2. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule;Aartsma-Rus;Muscle Nerve,2006
3. Systemic administration of PRO051 in Duchenne's muscular dystrophy;Goemans;N. Engl. J. Med.,2011
4. U7 snRNA: A tool for gene therapy;Gadgil;J. Gene Med.,2021
5. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells;Young;Cell Stem Cell,2016
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