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First proof of concept for a therapy to treat patients with NMNAT1-associated disease
September 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.

Read the article here: Gene therapy preserves retinal structure and function in a mouse model of NMNAT1-associated retinal degeneration

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