After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!
Tag Archive: Gene therapy
More than a decade of studies led by researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania culminated this week with the U.S. Food and Drug Administration’s approval of an innovative one-time gene therapy product indicated for the treatment of patients with a rare, inherited form of retinal blindness.
Your holiday season has been hectic, no doubt. Catch up with an early gift from us: Our biweekly roundup of research news from Children’s Hospital of Philadelphia comes with all the trimmings!
Buckle your seatbelts because this has been a busy week for research news at Children’s Hospital of Philadelphia.
Research at The Children’s Hospital of Philadelphia never stops. To help keep you informed about new discoveries and CHOP researchers’ views on timely topics, we are bringing together a roundup of news highlights. Look for this as a recurring feature here on Cornerstone.
When something important is missing, we often search for a replacement. After many years of looking, a team of researchers at The Children’s Hospital of Philadelphia and the University of Missouri have found a way to substitute for a missing gene linked to a relentless childhood neurodegenerative disease.
Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated animals with the bleeding disorder hemophilia.
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.
Investigators at The Children’s Hospital of Philadelphia are exploring a new gene therapy approach that aims to reactivate the production of fetal hemoglobin as a potential intervention for patients with sickle cell disease.
A recent Forbes article on the resurrection of the gene therapy business featured The Children’s Hospital of Philadelphia gene therapy expert Katherine A. High, MD.