A. INCREASE OUR UNDERSTANDING OF THE NEUROMUSCULAR SYSTEM
B. ENCOURAGE THE DEVELOPMENT OF THERAPIES FOR NEUROMUSCULAR DISEASES AND RARE GENETIC DISEASES
A. FUNDAMENTAL RESEARCH AND PHYSIOPATHOLOGY OF DISEASES OF THE NEUROMUSCULAR SYSTEM 1- Fundamental research projects aimed at increasing our comprehension of molecular, cellular, physiological and pathological mechanisms, involving the structure and function of the skeletal muscle, the motor neuron, the neuromuscular junction, aging and degeneration/regeneration in physiological and pathological conditions. 2- Research projects aimed at increasing our understanding of the clinical and genetic heterogeneity of neuromuscular diseases. 3- Understanding the physiopathology of smooth muscle in relation to neuromuscular diseases. 4-Differentiation of adult, embryonic and iPS stem cells into skeletal muscle or motor neurons (in physiological and pathological conditions). 5- Genetic/epigenetic regulation of genes and characterization of gene regulation networks within the neuromuscular system.
B. DEVELOPMENT OF THERAPEUTIC APPROACHES FOR RARE DISEASES 1/ Gene and/or cell therapy for rare disorders • Gene transfer and gene correction • Genetically modified cells • Development of delivery strategies • Control of the immune response (auto-immunity, anti-vector and anti-transgene responses, etc.) 2/ Strategies for modifying gene expression both at the gene and RNA level* • Projects based on gene transfer, chemical molecules or biotherapies *Concerning RNA as therapeutic target or as therapeutic product, please confer to the corresponding Call for Translational Research Projects (AFM-Telethon website) 3/ Pharmacotherapies of neuromuscular diseases The proposed projects must be clearly focused on neuromuscular diseases 4/ Translational research: tools for evaluation of treatments for neuromuscular diseases • Natural history of neuromuscular diseases • Outcome measures: functional, connect objects, imaging • Genomic, transcriptomic, proteomic, metabolomic biomarkers of pathologies and/or therapies • New cellular, tissue and animal models • Translational networking of basic, preclinical and clinical
http://www.afm-telethon.com/research/calls-for-proposals/current-calls-f...