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:
Author Keywords:
ryr1-rm
prime editing
ryr1 gene
crispr/cas9
protective mutation
calcium channel
calcium leak
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:
View Web of Science Record

Record Created: