Article

Improvement of TaC9-ABE mediated correction of human SMN2 gene

2024-03-23

Abstract excerpt

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by mutations in the survival motor neuron 1 ( SMN1 ) gene. Gene editing technology repairs the conversion of the 6th base T to C in exon 7 of the paralogous SMN2 gene, compensating for the SMN protein expression and promoting the survival and function of motor neurons. However, low editing efficiency and unintended off-target effects lim...

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Literature Corpus work: 4ae4dc54-3626-5ca8-992b-ad2759b34e47
DOI: 10.22541/au.171117510.02654435/v1

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