Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones and other health complications. For years, enzyme replacement therapy has been the only effective treatment for HPP. This therapy involves injecting asfotase alfa, an enzyme that replaces the missing one in patients’ bodies. However, these injections must be given three to six times a week, which can be challenging for many patients. While t

In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke (NINDS), a group of researchers have created a novel adeno-associated viral (AAV) vector to address brain disorders. This vector is more effective at reaching brain cells, opening doors for the development of treatment for brain disorders such as Parkinson's, Alzheimer's, and Huntington's Disease. Adeno-associated viral (AAV) vectors are common

New Approach for Liver Cancer Treatment and Possible Prevention: Gene Therapy Targets Biomarker for Hepatocellular Carcinoma (HCC) Researchers at the UC Davis Comprehensive Cancer Center have unveiled a promising gene therapy approach that significantly reduces the incidence of hepatocellular carcinoma (HCC) in animal models, specifically mice. This innovative research targets a specific protein known as galectin 1 (Gal1), which has long been established as a critical biomark