The U.S. National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $671,240 Project Grant (CFDA 93.846 Arthritis, Musculoskeletal and Skin Diseases Research) to the University of Washington to develop improved gene replacement and editing therapies for Duchenne muscular dystrophy (DMD). The objective is to address limitations of current DMD gene therapy approaches by refining technologies to express larger and more functional versions of the dystrophin protein,...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $585,000 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to the University of Iowa to investigate the role of the bone-regulating myokine FGF21 in Duchenne muscular dystrophy (DMD). The project aims to identify the cellular source and molecular mechanisms driving elevated FGF21 expression in dystrophic skeletal muscle, and to characterize the pathological effects of...
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 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 $525,048 to support the development and optimization of skeletal muscle delivery vehicles. The goal is to explore the use of muscle-specific cell fusogens, such as myomaker and myomerger, to engineer lentiviruses and extracellular vesicles that can selectively target and...
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), provides $303,065 to Kinea Bio Inc. to develop novel genetic medicine approaches to treat orphan diseases. The primary objectives are to demonstrate the feasibility of dual-AAV delivery of split dysferlin, evaluate the functionality of intein-generated dysferlin in preclinical models, and...
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), provides $306,872 to Vivreon Biosciences LLC to support the development of a novel small molecule calcium channel therapeutic to treat dystrophinopathies, including Duchenne muscular dystrophy (DMD). The key products and services to be delivered include:
Assessing the toxicity profile of the...
This $522,001 Project Grant awarded 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 to investigate the mechanisms by which the transcription factor DUX4 disrupts RNA metabolism in facioscapulohumeral muscular dystrophy (FSHD). The research aims to determine how DUX4 induces aberrant splicing and loss of RNA quality control, with the goal of better...
The University of California, Davis (UC Davis) was awarded a $123,424 Project Grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846). The grant supports research to elucidate the mechanisms by which fibrotic collagen architecture in skeletal muscle contributes to muscle stiffness and impairs muscle stem cell regenerative capacity. The project aims to assess the ability...
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 $102,938 to the University of California, Irvine (UC Irvine) to cultivate the next generation of muscle biology and muscular dystrophy (MD) researchers through a multidisciplinary training program.
The key objectives of this 5-year award, with a period of performance from May...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $531,109 Project Grant (CFDA 93.846) to the University of Florida's Division of Sponsored Research to develop technologies that enable safe and effective gene editing in skeletal muscle. The project aims to address key challenges in using adeno-associated virus (AAV) to deliver gene editing enzymes to skeletal muscle, including maximizing delivery and spread of the enzymes, avoiding sustained...