Timing of SMN replacement therapies in mouse models of spinal muscular atrophy: a systematic review and meta-analysis.

ABSTRACT: Mutations in the Survival of Motor Neuron 1 gene lead to a loss of survival motor neuron protein in patients with spinal muscular atrophy. Revolutionary advances in gene therapy have led to survival motor neuron-replacement therapies that significantly prolong life expectancy and improve neuromuscular function. However, accumulating evidence suggests that the timing of survival motor neuron-replacement therapies is a critical determinant of success. We performed a systematic review and meta-analysis of all pre-clinical studies testing survival motor neuron replacement therapies in mouse models of spinal muscular atrophy to assess the impact of timing of delivery on therapeutic effectiveness. We incorporated four databases in this pre-registered study (PROSPERO 2020 CRD42020200180):

SUBMITTER: Chaytow H 

PROVIDER: S-EPMC11342241 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature