Successful Gene Therapy Treatment and Redosing in an Animal Model of Methylmalonic Acidemia Petr O. Ilyinskii and Takashi Kei KishimotoSelecta Biosciences, Watertown, MA Gene therapy is making a difference in the lives of patients with previously untreatable genetic diseases. Most of

New Drug Relieves TCA Cycle Block In Patients With PA And MMA by: Jerry Vockley, M.D., PH.D. UNIVERSITY OF PITTSBURGHCLEVELAND FAMILY PROFESSOR OF PEDIATRIC RESEARCHPROFESSOR OF HUMAN GENETICS, CHILDREN’S HOSPITAL OF PITTSBURGHCHIEF OF MEDICAL GENETICS DIRECTOR OF THE CENTER FOR

Current Research Articles on Propionic Acidemia Major Breakthrough on Propionic Acidemia Research PaVe-GT: Paving the Way for Rare Disease Gene Therapies Adeno-associated virus serotype 8 (AAV8) Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia New Drug Relieves

​Effective Gene Therapy for Propionic Acidemia in Mice: One Organic Acidemia Helps Another Randy J. Chandler, Nuria Carrillo-Carrasco, and Charles P. Venditti  Organic Acid Research Section, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD  Email:venditti@mail.nih.gov For many