Researchers in the Center for Cellular and Molecular Therapeutics (CCMT) at The Children’s Hospital of Philadelphia suggest that an intricate pathway crucial to the development of Huntington’s disease (HD) rests on a “biological teeter-totter” that when carefully balanced could help to control this devastating neurodegenerative disorder.
Category Archive: Raymond G. Perelman Center for Cellular & Molecular Therapeutics
Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1.
High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA.
For her pioneering hemophilia and gene therapy research, Children’s Hospital hematologist Katherine A. High, MD, director of the Center for Cellular and Molecular Therapeutics (CCMT), was recently honored with the 2013 E. Donnall Thomas Lecture and Prize at the American Society of Hematology (ASH) Annual Meeting.
Gene therapy researchers at The Children’s Hospital of Philadelphia have produced a bioengineered decoy that by fooling the immune system prevents it from undermining the benefits delivered by a corrective gene.
A groundbreaking clinical trial of gene therapy for a form of congenital blindness, sponsored by The Children’s Hospital of Philadelphia in collaboration with Penn Medicine, was recently recognized with the Distinguished Clinical Research Achievement Award from the Clinical Research Forum, an organization of clinical research centers, industry, and volunteer groups.