Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel

Godbout, K., Dugas, M., Reiken, S. R., Ramezani, S., Falle, A., Rousseau, J., Wronska, A. E., Lamothe, G., Canet, G., Lu, Y., Planel, E., Marks, A. R., & Tremblay, J. P. (2025). Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel. International Journal of Molecular Sciences, 26(7), 2835. https://doi.org/10.3390/ijms26072835
Authors:
Kelly Godbout
Mathieu Dugas
Sina Ramezani
Alexia Falle
Gabriel Lamothe
Geoffrey Canet
Yaoyao Lu
Emmanuel Planel
Jacques P. Tremblay
Joel Rousseau
Steven R. Reiken
Anetta E. Wronska
Andrew R. Marks
Affiliated Authors:
Steven R. Reiken
Anetta E. Wronska
Andrew R. Marks
Columbia Affiliation:
Subjects:
Ryanodine Receptor Calcium Release Channel (MeSH)
Gene Editing (MeSH)
Muscular Diseases (MeSH)
Mutation (MeSH)
Genetic Therapy (MeSH)
Author Keywords:
calcium channel
calcium leak
crispr/cas9
prime editing
protective mutation
ryr1 gene
ryr1-rm
elink
Publication Type:
Article
Unique ID:
10.3390/ijms26072835
PMID:
40243436
DOI:
10.3390/ijms26072835
Journal:
International Journal of Molecular Sciences
Publication Date:
Data Source:
Web of Science
Source Link:
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Record Created: