Development and Characterization of a Modular CRISPR and RNA Aptamer Mediated Base Editing System

Juan Carlos Collantes, Victor M. Tan, Huiting Xu, Melany Ruiz-Uriguën, Amer Alasadi, Jingjing Guo, Hanlin Tao, Chi Su, Katarzyna M. Tyc, Tommaso Selmi, John J. Lambourne, Jennifer A. Harbottle, Jesse Stombaugh, Jinchuan Xing, Ceri M. Wiggins, Shengkan Jin

Producción científica: Contribución a una revistaArtículorevisión exhaustiva

7 Citas (Scopus)

Resumen

Conventional CRISPR approaches for precision genome editing rely on the introduction of DNA double-strand breaks (DSB) and activation of homology-directed repair (HDR), which is inherently genotoxic and inefficient in somatic cells. The development of base editing (BE) systems that edit a target base without requiring generation of DSB or HDR offers an alternative. Here, we describe a novel BE system called Pin-pointTM that recruits a DNA base-modifying enzyme through an RNA aptamer within the gRNA molecule. Pin-point is capable of efficiently modifying base pairs in the human genome with precision and low on-target indel formation. This system can potentially be applied for correcting pathogenic mutations, installing premature stop codons in pathological genes, and introducing other types of genetic changes for basic research and therapeutic development.

Idioma originalInglés
Páginas (desde-hasta)58-68
Número de páginas11
PublicaciónCRISPR Journal
Volumen4
N.º1
DOI
EstadoPublicada - feb. 2021
Publicado de forma externa

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