spinal muscular atrophyautosomal dominant childhood-onset proximal spinal muscular atrophy+1 moreResearch
A review highlights the potential of AAV9 vectors in gene therapy for inherited CNS diseases, building on the success of Zolgensma for spinal muscular atrophy. The International Society for Stem Cell Research provides a global map of marketed gene and cell therapies.
A similar expansion is underway with AAV9 vectors, which can cross the blood–brain barrier via intravascular delivery to treat inherited diseases of the CNS, as first demonstrated with Zolgensma for SMA. 196 , 197 , 198 , 199 , 200 For those interested in tracking marketed gene (and cell) therapies across the world, the International Society for Stem Cell Research maintains an up‐to‐date map.
Original title: “Clinical applications of gene therapy for rare diseases: A review - PMC”