The Dowling Lab

There are over 5000 rare genetic diseases, with less than 5% of these having approved treatments.  Nearly half of these diseases manifest in childhood, with 30% of affected children dying before age 5.  While genetic therapies have the potential to transform the treatment of rare diseases, a handful of success stories thus far focus on “n of 1” therapies designed to treat a specific patient mutations, leaving gene therapy inaccessible to the majority of rare genetic disease patients.  The Dowling Lab is passionate about developing the mechanistic insights, therapeutic tools and delivery vectors designed to combat these rare and devastating diseases for as many patients as possible.