Targeted-Deletion of a Tiny Sequence via Prime Editing to Restore SMN Expression
Spinal muscular atrophy (SMA) is a devastating autosomal recessive motor neuron disease associated with mutations in the survival motor neuron 1 (<i>SMN1</i>) gene, the leading genetic cause of infant mortality. A nearly identical copy gene (<i>SMN2</i>) is retained in almost...
Main Authors: | Miaojin Zhou, Shuqing Tang, Nannan Duan, Mi Xie, Zhuo Li, Mai Feng, Lingqian Wu, Zhiqing Hu, Desheng Liang |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2022-07-01
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Series: | International Journal of Molecular Sciences |
Subjects: | |
Online Access: | https://www.mdpi.com/1422-0067/23/14/7941 |
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