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

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...

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Bibliographic Details
Main Authors: Miaojin Zhou, Shuqing Tang, Nannan Duan, Mi Xie, Zhuo Li, Mai Feng, Lingqian Wu, Zhiqing Hu, Desheng Liang
Format: Article
Language:English
Published: MDPI AG 2022-07-01
Series:International Journal of Molecular Sciences
Subjects:
spinal muscular atrophy
survival motor neuron gene 2
intronic splicing silencer-N1
prime editing
targeted-deletion
induced pluripotent stem cells
Online Access:https://www.mdpi.com/1422-0067/23/14/7941

Internet

https://www.mdpi.com/1422-0067/23/14/7941

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