The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awarded a $395,537 Project Grant (CFDA 93.846 - Arthritis, Musculoskeletal and Skin Diseases Research) to Brigham & Women's Hospital, Inc. to develop a novel therapeutic strategy for treating osteopetrosis. The project will utilize CRISPR/Cas9 genome editing technology to correct osteopetrosis-causing CLCN7 gene mutations in hematopoietic progenitor cells, followed by autologous infusion of the corrected cells to restore normal bone remodeling in osteopetrotic mice. As a sub-award, Boston Children's Hospital will collaborate on designing and validating the CRISPR-based genome editing approaches. The grant aims to establish protocols for assessing, testing, and validating CRISPR/Cas9 technologies to provide a mutation-specific, cell-specific treatment for osteopetrosis, overcoming the limitations of current bone marrow transplantation therapies.
Generated 3/4/25, 7:51 AM