New molecular therapy restores vision in model of inherited childhood blindness — and may open the door to treatment for dozens of rare diseases
A University of Wisconsin–Madison team led by MERI RRF Daniel M. Albert Chair, Prof. Bikash Pattnaik, has developed a molecular therapy that restores vision in a mouse model of Leber congenital amaurosis type 16 (LCA16), a rare inherited childhood blindness with no currently approved treatment. How it works: Rather than editing DNA, the therapy uses …