RYR-1 Related Diseases With QA

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The RYR-1 Foundation is very excited to announce the recipients of the Foundation’s most recent round of research grants. These grants represent a commitment of over $270,000 of research funding. The awardees seek to apply cutting-edge, innovative therapeutic techniques to RYR-1-related diseases.

• Jocelyn Laporte, PhD, Institute of Genetics and Molecular and Cellular Biology (France): “Therapeutic proof-of-concepts for RYR1-related myopathy”
• Jacques Tremblay, PhD, Laval University (Canada): “PRIME editing correction of the T4709M mutation responsible for some cases of Ryanodine receptor type 1 (RYR1) related myopathies (RYR1 RM)”
• Susan Treves, PhD, and Francesco Zorzato, MD, PhD, University of Basel (Switzerland): “Treatment of an animal model carrying recessive RYR1 mutations with HDAC/DNA methyltransferase inhibitors”

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