International team led by Children’s National research scientists provides an explanation for why subtle cellular changes can result in dramatically different genetic disorders Read our press releaseRead about cell research
Eric Hoffman, PhD earned his degree in Genetics at Johns Hopkins University and then pursued post-doctoral training with Louis Kunkel at Boston Children’s Hospital working on Duchenne muscular dystrophy gene and protein identification. Dr. Hoffman’s research has focused on molecular genetics, pathogenesis, and therapeutics of neuromuscular disease. The research group he directs at Children’s National hosts research laboratories of faculty focusing on the ‘omics applications to pediatric health problems.
Recent areas of particular interest to Dr. Hoffman and his laboratory include the development of therapeutics for Duchenne muscular dystrophy and furthering our understanding of the molecular genetics of muscle in health and disease. Relevant to drug development, Dr. Hoffman co-founded ReveraGen Biopharma, where a lead dissociative steroid (VBP15) is being developed for muscular dystrophy and other disorders. VBP15 development is funded by Foundation to Eradicate Duchenne (FED), the NIH TRND program, and Muscular Dystrophy Association Venture Philanthropy. Dr. Hoffman has been assisting in the development of systemic oligonucleotide therapeutics (exon skipping), in collaboration with AVI and other academic laboratories. Dr. Hoffman directs a Program Project on exon skipping from the Department of Defense, with research projects on small molecules to enhance potency of oligonucleotide drugs (Carrie Miceli, UCLA), oligo conjugates to increase cell delivery (Qi Lu, Carolinas Medical Center), and large animal studies of efficacy (Toshifumi Yokota and Shin’ichi Takeda, Tokyo), with a pre-clinical testing core (Kanneboyina Nagaraju, Childran’s National). He is Co-Director of a NIAMS Center of Research Translation (P50 CORT) with Paula Clemens (Pittsburgh), overseeing projects on studies of antisense ‘skipped’ dystrophin protein function, optimization of multiple morpholino drugs, and an international natural history study of Becker muscular dystrophy. He helps direct a U54 Center of Pediatric Pharmacology focused on studies of chronic kidney toxicity with long-term morpholino oligonucleotide treatment (with Drs. John Van den Anker and Edward Connor from Children’s National).
He previously served as PI on GLP toxicity studies of morpholinos, supported by the Department of Defense that enabled U.S. trials of high dose IV morpholino in Duchenne muscular dystrophy. Dr. Hoffman serves as Scientific Director of the 24 site Cooperative International Neuromuscular Research Group (CINRG) and as PI of a NIH National Center for Medical Rehabilitation Research. Dr. Hoffman is board-certified by the American Board of Medical Genetics in Clinical Molecular Genetics.