Gene therapies to halt the manifestation and/or progression of devastating diseases such as spinal muscular atrophy (AAV9-SMN1, Zolgensma®, Novartis) and biallelic RPE65 mutation-associated retinal dystrophy (AAV2-RPE65, Luxturna®, Spark Therapeutics) have been at the forefront of medical advances and their recent approval by regulatory institutions paves the way for the use of gene delivery-based regenerative methodologies in other settings. Here, RPE65 is linked to spinal muscular atrophy.