Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development. Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate
Heart disease remains the leading cause of death in the world. One reason is that unlike other tissues, such as bone and skin, the heart has remarkably poor regenerative capability after an injury such as a heart attack. Scientists have therefore searched for heart cells that have regenerative properties. A new study by the Yoshinori
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency. “These results confirm the stability and good
A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides
Two patients represent longest-known CAR T cell response to date, providing insight into treatment effects and outcomes In the summer of 2010, Bill Ludwig and Doug Olson were battling an insidious blood cancer called chronic lymphocytic leukemia (CLL). They’d both received numerous treatments, and as remaining options became scarce, they volunteered to become the first
Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient, and predictable gene and cell therapies. Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and
Cancer cells increase the synthesis of sugars, that once exposed on the surface create a “shield” capable of hindering the immune system. According to a study published today in Science Translational Medicine, this shield could explain, at least in part, the reduced efficacy of CAR T cell therapies in solid tumors. The researchers also found a way to
An investigational gene therapy showed benefit in pediatric patients with early-onset metachromatic leukodystrophy (MLD), a small study showed. Treatment with atidarsagene autotemcel (arsa-cel) preserved motor development and cognitive function in MLD patients enrolled in a prospective phase I/II trial or treated through expanded access programs, reported Alessandro Aiuti, MD, of the San Raffaele Telethon Institute for Gene
Peripheral nerve injury (PNI) refers to varying degrees of trauma to peripheral nerve stems or branches. PNIaccounts for 1.5–4.0% of global trauma cases annually and is one of the most challenging health issues at present. Nerve regeneration is a complicated cellular process involving infammation, neurotrophic factors, neurotransmitters, adhesion, the formation of axons and growth cones,
Research from the School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused by a mutation in the SCN1A gene, resulting in
