Gene Therapy Reverses Effects of SMARD1 in a mouse model

Gene Therapy Reverses Effects of SMARD1 in a mouse model

A study conducted by Centro Dino Ferrari, Università degli Studi di Milano and IRCCS Fondazione Ca’ Granda Ospedale Maggiore Policlinico – published in Science Advances – (lead authors Monica Nizzardo and Stefania Corti) describes the efficacy of AAV9 gene therapy in the treatment of Spinal Muscular Atrophy with respiratory distress type 1.

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