Visiting Speaker: Zarife Sahenk, MD, PhD

Zarife Sahenk, MD, PhD’s laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children's Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory.