by Miguel Sena-Esteves, The Conversation Credit: Pixabay/CC0 Public Domain 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...
by Melissa Rohman, Northwestern University Graphical abstract. Credit: DOI: 10.1056/NEJMoa2113206 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...
By MANSUR SHAHEEN U.S. DEPUTY HEALTH EDITOR FOR DAILYMAIL.COM and ASSOCIATED PRESS PUBLISHED: 15:21 EST, 2 February 2022 | UPDATED: 15:27 EST, 2 February 2022 An experimental gene therapy treatment that transforms a person’s blood cells into cancer-killers could be a cure for leukemia. Researchers at the University of Pennsylvania performed CAR-T cell therapy treatment on two patients who were suffering from...
MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS IMAGE: THE FIRST PEER-REVIEWED JOURNAL IN THE FIELD AND PROVIDES ALL-INCLUSIVE ACCESS TO THE CRITICAL PILLARS OF HUMAN GENE THERAPY: RESEARCH, METHODS, AND CLINICAL APPLICATIONS. CREDIT: MARY ANN LIEBERT INC., PUBLISHERS Gene therapy targeting the messenger RNA (mRNA) of the mutated huntingtin gene (HTT) can provide long-lasting therapeutic benefit...
by Josh Barney, University of Virginia Credit: CC0 Public Domain Research from the University of Virginia 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...
A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients. Four patients at Columbia University Irving Medical Center/NewYork-Presbyterian participated in the multicenter study, the first to...
by Children’s Hospital Boston This scanning electron micrograph shows sensory outer hair cells, which are required for cochlear amplification and normal hearing. The image at left shows a disorganized sensory hair cell from the inner ear from a mouse with a mutation in the Strc gene; as a result, the cell is missing scaffolding links...
Broad and robust transgene expression across brain regions of marmoset is shown after systemic delivery with engineered capsid. Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington’s disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into...
by Lori Dajose, California Institute of Technology Multiple sites of the AAV capsid are engineered to acquire brain specificity while being targeted away from the liver in mice after in vivo selection. On the left, the engineering sites on the AAV capsid and monomer are depicted in purple and green, while on the right transgene...
AFM-TÉLÉTHON PARIS, FRANCE (November xx, 2021) – Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today publication in the open-access journal Science Advances of research describing advances in using AAV-based gene therapy for potential long-lasting treatment of X-linked hypophosphatemia (XLH), a serious skeletal disease caused by low levels of phosphate...
