CRISPR artificial splicing factors

Menghan Du, Nathaniel Jillette, Jacqueline Jufen Zhu, Sheng Li and Albert Wu Cheng ()
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Menghan Du: The Jackson Laboratory for Genomic Medicine
Nathaniel Jillette: The Jackson Laboratory for Genomic Medicine
Jacqueline Jufen Zhu: The Jackson Laboratory for Genomic Medicine
Sheng Li: The Jackson Laboratory for Genomic Medicine
Albert Wu Cheng: The Jackson Laboratory for Genomic Medicine

Nature Communications, 2020, vol. 11, issue 1, 1-11

Abstract: Abstract Alternative splicing allows expression of mRNA isoforms from a single gene, expanding the diversity of the proteome. Its prevalence in normal biological and disease processes warrant precise tools for modulation. Here we report the engineering of CRISPR Artificial Splicing Factors (CASFx) based on RNA-targeting CRISPR-Cas systems. We show that simultaneous exon inclusion and exclusion can be induced at distinct targets by differential positioning of CASFx. We also create inducible CASFx (iCASFx) using the FKBP-FRB chemical-inducible dimerization domain, allowing small molecule control of alternative splicing. Finally, we demonstrate the activation of SMN2 exon 7 splicing in spinal muscular atrophy (SMA) patient fibroblasts, suggesting a potential application of the CASFx system.

Date: 2020
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DOI: 10.1038/s41467-020-16806-4

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