The labs of Artur A. Indzhykulian, MD, PhD, Assistant Professor of Otolaryngology–Head and Neck Surgery at Harvard Medical School (HMS), and David Corey, PhD, Bertarelli Professor of Translational Medical Science in the Blavatnik Institute of HMS, have recently received a $1 million Blavatnik Therapeutics Challenge Award for their collaborative research on the treatment of Usher syndrome 1F, a rare – albeit debilitating – form of Usher syndrome.
The Blavatnik Therapeutics Challenge Awards are designed to accelerate basic science advances into new, frontline therapeutics, according to HMS. Now in its second year of existence, the awards are made possible thanks to a transformative gift from the Blavatnik Family Foundation, which provides award recipients with $1 million over two years.
Usher syndrome 1F is a rare hereditary disease that results in profound deafness and lack of balance at birth, and blindness that progresses over time. Mutations to a single gene are responsible for the disease. Dr. Corey, principal investigator of the award-winning project, will use the funds to collaborate with Dr. Indzhykulian, who also serves as an assistant scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear, on developing a gene-therapy technique that would restore sensory function. The technique will aim to shrink a copy of the missing gene into a size small enough to fit inside a vector, which would then replace the faulty gene with the shortened copy.