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 $395,537 to Brigham & Women's Hospital Inc. to develop and validate CRISPR-based genome editing strategies to correct genetic mutations in the CLCN7 gene that cause osteopetrosis, a rare and debilitating skeletal disorder. The project aims to optimize genome editing techniques in...
This $159,594 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), supports research to identify new therapeutic targets for osteogenesis imperfecta (OI), a rare genetic bone disorder. The award recipient, the Research Foundation of the City University of New York (RFCUNY), will investigate the intracellular signaling and extracellular matrix mechanisms...
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 $386,481 to the University of Connecticut Health Center to conduct research on cellular therapy for osteogenesis imperfecta (OI). The key objectives are to evaluate different cell populations as progenitor sources, determine the mechanisms by which irradiation improves cell engraftment, 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), 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, with a total funding amount of $454,050.00, was 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). The project aims to develop a novel, adeno-associated virus (AAV)-based gene therapy for healing critical size, osseous defects. Key objectives include: 1) evaluating the level and duration of transgene expression using an AAV8-RGD...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $123,040 Project Grant under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to the University of Kansas Medical Center Research Institute, Inc. (KUMCRI) to investigate the therapeutic role of the NFAT1 transcription factor in osteoarthritis. The 5-year project, which began on August 1, 2024, aims to evaluate the potential of AAV-delivered NFAT1 gene therapy in treating...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded Vanderbilt University a $393,412 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to develop engineered cells as agents for treating osteoarthritis. The project aims to leverage advanced cell design platforms to create synthetic receptor-controlled, joint-resident cells that can detect cartilage degeneration and then upregulate the expression of transgenes to promote...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $1,066,302 Project Grant (CFDA 93.846) to the University of Massachusetts Medical School (UMass Medical) to create a comprehensive biomedical knowledgebase for the Osteoarthritis Initiative (OAI) cohort study. The goal is to promote and facilitate the use of the OAI's extensive clinical data, medical images, genetics, and biospecimens to accelerate osteoarthritis research. Key products and services...
This $589,255 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 small RNA targeting approaches, such as microRNA and siRNA, to advance treatments for heterotopic ossification (HO). HO is a condition involving pathologic bone formation in soft tissues that can occur following orthopedic surgeries or injuries. The award will fund the...
This $313,000 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) will fund research by Emory University to establish phosphorylated WNT5A produced by osteoclasts as a potential anabolic bone drug. The research aims to determine how osteoclast-derived phospho-WNT5A can enhance bone formation in vivo and elucidate the cellular and molecular mechanisms by...
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $221,100 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to the University of Massachusetts Medical School to develop and evaluate in vivo gene editing strategies to repair collagen mutations in mouse models of osteogenesis imperfecta (OI). The 2-year project aims to use bone-targeting recombinant adeno-associated virus (rAAV) to implement precise gene editing techniques, such as CRISPR-Cas9 nickase (prime editing), Cas12A, and homology-directed repair boosting, to correct the genetic defects underlying OI without off-target effects. Successful completion of this research would provide proof-of-concept for a novel gene therapy platform to treat OI and potentially other skeletal diseases.