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Article

Targeted-Deletion of a Tiny Sequence via Prime Editing to Restore SMN Expression

19 Jul 2022

Abstract excerpt

Spinal muscular atrophy (SMA) is a devastating autosomal recessive motor neuron disease associated with mutations in the survival motor neuron 1 (SMN1) gene, the leading genetic cause of infant mortality. A nearly identical copy gene (SMN2) is retained in almost all patients with SMA. However, SMN2 fails to prevent disease development because of its alternative splicing, leading to a lack of exon 7 in the...

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PMID: 35887289
DOI: 10.3390/ijms23147941

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