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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 (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...

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Detalles Bibliográficos
Autores principales:	Zhou, Miaojin, Tang, Shuqing, Duan, Nannan, Xie, Mi, Li, Zhuo, Feng, Mai, Wu, Lingqian, Hu, Zhiqing, Liang, Desheng
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9317564/ https://www.ncbi.nlm.nih.gov/pubmed/35887289 http://dx.doi.org/10.3390/ijms23147941

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