The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $295,294 Phase I SBIR project grant (CFDA 93.846) to Gigamune, Inc. to develop a novel lentiviral gene delivery platform for in vivo delivery of the full-length dystrophin gene to muscle cells. The goal is to create an efficient and targeted approach for gene therapy to treat Duchenne muscular dystrophy, a severe genetic disorder causing muscle wasting. The project aims to leverage Gigamune's...
This Project Grant award of $2,123,500.00 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) aims to develop a novel gene therapy approach to treat muscular dystrophies. The key products and services to be delivered under this grant include: Demonstrating the feasibility of using a dual adeno-associated viral (AAV) vector system to deliver full-length dysferlin, a protein deficient in...
This Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), aims to develop enabling technologies for safe and effective gene editing in skeletal muscle. The $531,109 grant awarded to the University of Florida will fund research to address key challenges in applying gene editing to skeletal muscle diseases, including maximizing delivery and spread of editing...
This federal Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) aims to develop improved gene replacement and editing vectors for the treatment of Duchenne muscular dystrophy (DMD). The $671,240 award will fund research at the University of Washington over a 5-year period from January 2025 to December 2029. The key objectives are to: 1) Refine technologies...
This $1,557,819 Project Grant award from the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research) supports muscular dystrophy research at the Research Institute at Nationwide Children's Hospital in Columbus, Ohio. The award funds three key research projects: Project 1 (PI: Paul Martin, PhD) aims to utilize a novel bicistronic AAV vector to both prevent muscle disease and build new muscle function in patients with limb...
This Project Grant award of $473,377 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research, supports research to modulate the stem cell niche and improve skeletal muscle regeneration. The key objectives are to: Interrogate how muscle cells arise and develop functional characteristics from human pluripotent stem cells (hPSCs) through analysis of myogenic commitment factors and inhibition of...
This federal Project Grant award of $692,292.00 from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program supports research to understand the regulation of pediatric skeletal muscle regeneration and its implications for treating muscle diseases such as Duchenne muscular dystrophy (DMD). The research aims to examine the role of the muscle niche, including immune and...
This federal Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), provides $151,471.00 to the University of California, Davis to conduct research on using collagenase to target collagen architecture in fibrotic skeletal muscle. The goal is to reduce muscle stiffness and promote tissue regeneration, particularly in the context of Duchenne muscular...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $647,460 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to Cornell University to support research on the mechanisms of mechanically-induced gene expression in skeletal muscle cells. The five-year project, running from September 1, 2024 to August 31, 2029, aims to define the nuclear mechanisms responsible for the ultra-rapid activation of mechanoresponsive genes in...
This federal Project Grant award, totaling $1,601,933 and provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846), supports research into therapeutic approaches for muscular dystrophy caused by mutations in the dysferlin gene (DYSF). The primary awardee, the University of Maryland, Baltimore, is conducting research to optimize engineered constructs of the DYSF-C2A domain...