Bone & Muscle Diseases & Injuries

Musculoskeletal disorders and injuries affect bones, muscles, joints and connective tissues. They can lead to pain and loss of function and are typically characterized by limitations in mobility and dexterity, which greatly impact people’s quality of life and ability to work. Our scientists are using stem cells Cells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves. stem cells Cells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves. to develop cell replacement and gene editing A type of gene therapy that works by delivering genetic material that can directly edit pieces of DNA within a cell. This changes the instructions the DNA encodes for, which ultimately results in an increase or decrease in the production of a certain protein and the restoration of proper cell function. gene editing A type of gene therapy that works by delivering genetic material that can directly edit pieces of DNA within a cell. This changes the instructions the DNA encodes for, which ultimately results in an increase or decrease in the production of a certain protein and the restoration of proper cell function. therapies to regenerate damaged bones and muscles and — in some cases — prevent this damage from occurring. This includes correcting the genetic defects that cause muscular dystrophies like Duchenne and rejuvenating aging muscle stem cells. By pursuing strategies to keep stem cells functionally young, researchers are inching closer to developing therapies for seniors that allow for speedier and less challenging recovery from bone fractures or muscle injuries.

Center investigators have developed an immunotherapy A type of treatment that uses the body's own immune system to fight cancer, infections and other diseases. This approach has revolutionized cancer care and is also being applied in experimental treatments for HIV, lupus and other conditions. immunotherapy A type of treatment that uses the body's own immune system to fight cancer, infections and other diseases. This approach has revolutionized cancer care and is also being applied in experimental treatments for HIV, lupus and other conditions. to treat metastatic sarcoma, a group of notoriously hard-to-treat cancers that occur in the bones and connective tissue, such as fat and muscle. They’ve also engineered a bone-building drug that, when administered in mice aboard the International Space Station, has prevented the bone loss that commonly occurs during long-duration space travel. The success of this study not only points to a promising therapy for astronauts, but also for patients on Earth who experience bone loss due to aging and conditions like osteoporosis.