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Updated: Feb 26, 2026

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Edición de bases de adenina concentrada espacialmente para corregir eficientemente mutaciones de PLP1 en

Chi Zhang1, Ke Ye1, Yafang Shang2

  • 1Department of Histoembryology, Genetics and Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.

Nucleic acids research
|February 25, 2026
PubMed
Resumen

Desarrollamos un editor de bases de adenina concentrado espacialmente (cABE) para mejorar la edición génica en oligodendrocitos (OLs). Esta estrategia mejora la corrección de mutaciones que causan la enfermedad de Pelizaeus-Merzbacher (PMD), ofreciendo una terapia potencial para este trastorno de la mielina.

Palabras clave:
edición génicaoligodendrocitosenfermedad de Pelizaeus-Merzbachertrastornos de la mielinaterapia génica

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