Successful Correction by Prime Editing of a Mutation in the <i>RYR1</i> Gene Responsible for a Myopathy

Successful Correction by Prime Editing of a Mutation in the <i>RYR1</i> Gene Responsible for a Myopathy

We report the first correction from prime editing a mutation in the <i>RYR1</i> gene, paving the way to gene therapies for RYR1-related myopathies. The <i>RYR1</i> gene codes for a calcium channel named Ryanodine receptor 1, which is expressed in skeletal muscle fibers. The f...

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Bibliographic Details
Main Authors: Kelly Godbout, Joël Rousseau, Jacques P. Tremblay
Format: Article
Language:English
Published: MDPI AG 2023-12-01
Series:Cells
Subjects:
prime editing
CRISPR/Cas9
gene editing
gene therapy
RYR1-related diseases
neuromuscular diseases
Online Access:https://www.mdpi.com/2073-4409/13/1/31

Internet

https://www.mdpi.com/2073-4409/13/1/31

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