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CRISPR artificial splicing factors

Author

Listed:
  • Menghan Du

    (The Jackson Laboratory for Genomic Medicine
    University of Connecticut Health Center)

  • 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
    University of Connecticut Health Center
    The Jackson Laboratory Cancer Center
    University of Connecticut)

  • Albert Wu Cheng

    (The Jackson Laboratory for Genomic Medicine
    University of Connecticut Health Center
    The Jackson Laboratory Cancer Center
    University of Connecticut Health Center)

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.

Suggested Citation

  • Menghan Du & Nathaniel Jillette & Jacqueline Jufen Zhu & Sheng Li & Albert Wu Cheng, 2020. "CRISPR artificial splicing factors," Nature Communications, Nature, vol. 11(1), pages 1-11, December.
    • Handle: RePEc:nat:natcom:v:11:y:2020:i:1:d:10.1038_s41467-020-16806-4
    DOI: 10.1038/s41467-020-16806-4
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    Cited by:

    1. Shailendra Kumar & Sanghamitra Choudhury, 2022. "Gender and feminist considerations in artificial intelligence from a developing-world perspective, with India as a case study," Palgrave Communications, Palgrave Macmillan, vol. 9(1), pages 1-9, December.

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