Category: Research Updates

A new publication from Dr. Melissa Spencer’s laboratory at the University of California Los Angeles identifies a compound that mimics the effects of exercise in a mouse model of limb girdle muscular dystrophy type 2A (LGMD2A/R1). These studies may point the way to similar compounds that will benefit people living with muscular dystrophy.    Mutations…

PUBLISHED RESEARCH ALERT: Plasmid-Mediated Gene Therapy in Mouse Models of LGMD Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of a new research paper from the group of Dr. Michele Calos, Stanford University School of Medicine. The paper, titled “Plasmid-mediated gene therapy in mouse models of LGMD,” was published in Molecular Therapy:…

Published research alert: Titin splicing regulates cardiotoxicity associated with Calpain-3 gene therapy for LGMD2A/R1 Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of research undertaken by Dr. Isabelle Richard, Head of the Progressive Dystrophy Laboratory, Généthon, and colleagues. The paper, titled “Titin splicing regulates cardiotoxicity associated with Calpain-3 gene therapy for LGMD2A/R1,”…

Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of important research undertaken by Dr. Rita Perlingeiro, Professor of Medicine, University of Minnesota, and colleagues. The paper, titled “Gene Correction of LGMD2A Specific iPSCs for the Development of Targeted Autologous Cell Therapy,” was published in Molecular Therapy.  The group used the technique CRISPR-Cas9 to correct…

C3 is excited to announce that we are funding a research project with Dr. Cathleen Lutz, Director of the Rare and Orphan Disease Center at The Jackson Laboratory in Bar Harbor, Maine. Dr. Lutz works with researchers and disease foundations in the development, characterization and validation of mouse models. These models can then be distributed…