First proof of concept for a therapy to treat patients with NMNAT1-associated diseaseSeptember 16, 2020
Molecular Therapy: Methods & Clinical Development Vol. 18 September 2020 ª 2020
Authors from the Ocular Genomics Institute and Grousebeck Gene therapy Center have published the first proof of concept for a therapy to treat patients with NMNAT1-associated disease. No treatment is available for nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1)-associated retinal degeneration, an inherited disease that leads to severe vision loss early in life.