Less is more: Allele-specific removal of dysfunctional RYR1 channel subunits-Reference-Cited by-同舟云学术

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Less is more: Allele-specific removal of dysfunctional RYR1 channel subunits

Published:2024-09 Issue:3 Volume:35 Page:102301
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Sun Derek,Lagor William R.^ORCID

Publisher

Elsevier BV

Reference5 articles.

1. Functional benefit of CRISPR-Cas9-induced allele deletion for RYR1 dominant mutation;Beaufils;Mol. Ther. Nucleic Acids,2024

2. Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy;Godbout;Cells,2023

3. Allele-specific gene silencing in two mouse models of autosomal dominant skeletal myopathy;Loy;PLoS One,2012

4. An autosomal dominant congenital myopathy with cores and rods is associated with a neomutation in the RYR1 gene encoding the skeletal muscle ryanodine receptor;Monnier;Hum. Mol. Genet.,2000

5. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models;Hakim;Nat. Commun.,2021

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