Article

Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing

2023-04-06

Abstract excerpt

Defect in the SMN1 gene causes spinal muscular atrophy (SMA), which shows loss of motor nerve cells, muscle weakness and atrophy. While current treatment strategies, including small molecules or viral vectors, have been reported to improve motor function and survival, an ultimate and long-term treatment to correct SMA endogenous mutations and improve its phenotypes is still highly challenging. We have previously...

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Literature Corpus work: 4e877add-7424-5471-a008-63d3a7072526
DOI: 10.1101/2023.04.06.535786

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