Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy

RNA-Based Therapeutics: Current Progress and Future Prospects

(2012) John C. Burnett et al.   CHEMISTRY & BIOLOGY

AICAR prevents heat-induced sudden death in RyR1 mutant mice independent of AMPK activation

(2012) Johanna T Lanner et al.   NATURE MEDICINE

Current progress of siRNA/shRNA therapeutics in clinical trials

(2011) John C. Burnett et al.   Biotechnology Journal

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

The I4895T mutation in the type 1 ryanodine receptor induces fiber-type specific alterations in skeletal muscle that mimic premature aging

(2010) Simona Boncompagni et al.   AGING CELL

Muscle weakness inRyr1I4895T/WTknock-in mice as a result of reduced ryanodine receptor Ca2+ion permeation and release from the sarcoplasmic reticulum

(2010) Ryan E. Loy et al.   JOURNAL OF GENERAL PHYSIOLOGY

RNAi-based therapeutics-current status, challenges and prospects

(2009) Katrin Tiemann et al.   EMBO Molecular Medicine

A retrograde signal from RyR1 alters DHP receptor inactivation and limits window Ca2+ release in muscle fibers of Y522S RyR1 knock-in mice

(2009) Z. Andronache et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

(2009) T. M. Wheeler et al.   SCIENCE

RyR1 S-Nitrosylation Underlies Environmental Heat Stroke and Sudden Death in Y522S RyR1 Knockin Mice

(2008) William J. Durham et al.   CELL