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 conditions. Several sessions were dedicated to muscular dystrophies, and four presentations focused on animal and…
Read MoreDufour Lab to investigate the biological role of calpain 3 in muscle
Coalition to Cure Calpain 3 (C3) is celebrating the New Year with a new research grant! We are pleased to announce a grant has been awarded to Dr. Antoine Dufour, Assistant Professor of Physiology and Pharmacology at the University of Calgary. The project, titled “Unbiased Systems-wide Investigation of Calpain 3 in Patients with LGMD2A/R1 Using Mass Spectrometry,”…
Read MorePlasmid-mediated Gene Therapy Shows Promise for LGMD2A/R1 Patients
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:…
Read MorePromising 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, and colleagues. The paper, titled “Titin splicing regulates cardiotoxicity associated with Calpain-3 gene therapy for LGMD2A/R1,”…
Read MoreC3 Scientific Director Dr. Jennifer Levy Appointed to Muscular Dystrophy Coordinating Committee
The Muscular Dystrophy Coordinating Committee (MDCC) is a federal advisory committee that aims to coordinate the activities repeated to muscular dystrophy research across the National Institutes of Health (NIH) and other federal programs including the Centers for Disease Control and Prevention, the Food and Drug Administration, and the Department of Defense. The 18 members of the MDCC…
Read More2019 World Muscle Society Recap
The 24th International Annual Congress of the World Muscle Society was held October 1-5, 2019 in Copenhagen, Denmark. C3 Scientific Director Dr. Jennifer Levy represented Coalition to Cure Calpain 3 (C3) and presented a poster detailing the C3 mission, patient registry, and grant programs. The Congress covered a variety of topics related to advances across…
Read MoreAn LGMD by any other name… is still an LGMD: New nomenclature for the LGMDs
Those who recently attended the National LGMD Conference in Chicago learned that the LGMDs are getting new names. In the old naming system, dominant LGMDs were called LGMD1s and recessive LGMDs were called LGMD2s. The subtypes were further delineated by a letter to designate the gene that contained the disease-causing mutation(s). In the case of…
Read MorePublished 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 Cell Therapy,” was published in Molecular Therapy. The group used the technique CRISPR-Cas9 to correct…
Read MoreDr. Stefanie Müthel Awarded First Early Career Investigator Travel Grant
Coalition to Cure Calpain 3 (C3) is pleased to announce that Dr. Stefanie Müthel was awarded a Travel Grant for the presentation of her research at the Biology of Calpains in Health and Disease Conference in Pacific Grove, California, from July 14-19, 2019. Dr. Müthel is a postdoctoral researcher in the laboratory of Dr. Simone Spuler at…
Read MoreDefining Clinical Endpoints in LGMD
Coalition to Cure Calpain 3 (C3) is pleased to announce that a research grant has been awarded to Dr. Nicholas Johnson, Associate Professor & Vice Chair of Research, Virginia Commonwealth University. The project is titled “Defining Clinical Endpoints in LGMD.” What are COAs, and why do they matter to those living with LGMD? Clinical outcome assessments (COAs)…
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