Disease models

New LGMD2A/R1 mouse model available at Jackson Labs

Coalition to Cure Calpain 3 (C3) is pleased to share that a new Calpain 3-null mouse strain is available through the Jackson Labs repository. This line is crossed onto an NSG background, resulting in an immunodeficient strain. The NSG.Capn3 KO mice may

Publication shares development of new animal model of LGMD2A/R1

A paper published by Jason Berman, Children’s Hospital of Eastern Ontario Research Institute and University of Ottawa, along with colleagues from Dalhousie University, AGADA Biosciences, and Binghamton University – State University of New York, shares the development of a zebrafish

New LGMD2A/R1 Mice: Novel tools for drug development

Coalition to Cure Calpain 3 (C3) is excited to announce the generation of four mouse models of limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These mouse models were developed and characterized as part of a research grant from C3 to Dr.

C3 funds development of new LGMD2A/R1 disease model for preclinical testing

Coalition to Cure Calpain 3 (C3) is pleased to announce the funding of a new research award to Dr. Pia Elustondo of AGADA Biosciences. This project will characterize a novel LGMD2A/R1 disease model and utilize this model to test several

Published Research Alert: CAPN3 gene therapy improves muscles function in a mouse model of limb-girdle muscular dystrophy type 2A/R1 (LGMD2A/R1)

Coalition to Cure Calpain 3 (C3) is pleased to announce the publication of important research undertaken by Dr. Zarife Sahenk, Dr. Jerry Mendell, and colleagues at Nationwide Children’s Hospital in Columbus, Ohio. The paper, titled “Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the

Notes from 23rd annual meeting of the American Society for Gene and Cell Therapy

The American Society for Gene and Cell Therapy (ASGCT) annual meeting kicked off on Tuesday, May 12, 2020. Due to COVID-19, the meeting was held virtually. This meeting covers the field’s latest and most innovative science across all diseases and

Promising Insights into Safety of LGMD2A/R1 Gene Therapy Approach

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,

Published Research Alert: Gene Correction of LGMD2A Specific iPSCs for the Development of Targeted Autologous Cell Therapy

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

C3 supports generation of new LGMD2A disease model for research

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