Job title: Senior Investigator
Carsten Bönnemann graduated from Medical School at the University of Freiburg in Germany. He trained in pediatrics in Hamburg and Göttingen (where he was awarded the habilitation/venia legendi in pediatrics), and in neurology/child neurology at Massachusetts General Hospital/Harvard Medical School in Boston. He did postdoctoral research in genetics and neuromuscular specialty training at Boston Children’s Hospital/Harvard Medical School. From 2002 he was on the faculty as Co-Director of the Neuromuscular Program and Director of the Pediatric Neurogenetics Clinic at Children’s Hospital of Philadelphia/University of Pennsylvania School of Medicine, where he continues to be on faculty as Adjunct Full Professor of Neurology. In 2010 he was recruited to the NIH as a tenured Senior Investigator and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section in the Neurogenetics Branch of the National Institute of Neurological Disorders and Stroke. Clinical, genomic and translational work in the Section centers in particular around early onset neuromuscular disorders such as the congenital myopathies and congenital muscular dystrophies and on the development of molecular and gene directed treatment approaches to these conditions, including first-in-human intrathecal and intravenous AAV mediated gene transfer trials. Dr. Bönnemann was a Pew Fellow in the Biomedical Sciences and in 2010 received the Derek Denny-Brown Neurological Scholar Award from the American Neurological Association. He is Co-Editor-in-Chief of the Journal of Neuromuscular Diseases (JND). He has authored more than 275 papers, reviews and chapters in the field of pediatric neuromuscular and neurogenetic disorders.
Live Q&A – Ask the speakers your questions 10:20 am
day: Day One
Intrathecal Gene Therapy for Giant Axonal Neuropathy: Immunological Considerations 9:30 am
• A case study exploring the first in human intrathecal delivery of AAV9 gene therapy for CNS targeting • Formulating an immuno-modulatory strategy to mitigate the innate, anticapsid & anti-transgene immune response after intrathecal delivery • Exploring how immune modulation strategies can be translated to other indication areas and routes of gene therapy deliveryRead more
day: Day One