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RNA mis-splicing in disease

Scotti, Marina M., Swanson, Maurice S.·2016·Nature Reviews Genetics
DOI 10.1038/nrg.2015.3PMC PMC5993438
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  • Cas13a can target pre-mRNA, which can be useful in diseases caused by mis-splicing.

    “Cas13a can also target pre-mRNA, which can be useful in diseases caused by mis-splicing [126], since the enzyme can act before the defective splicing occurs.”

    From CRISPR-Cas: Converting A Bacterial Defence Mechanism into A State-of-the-Art Genetic Manipulation Tool (2019) · cites this paper as [126]

    The “Therapies to modulate RNA mis-splicing” section (page unavailable) describes targeting nascent transcripts as a therapeutic approach for diseases involving mis-splicing. It discusses antisense oligonucleotides, not Cas13a, and does not establish that Cas13a targets pre-mRNA or acts before defective splicing occurs. (LLM, conf=0.84)

    Experimental — accuracy not yet measured

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Metadata

Authors
Scotti, M. M., & Swanson, M. S.
Venue
Nature Reviews Genetics
Publisher
Springer Science and Business Media LLC
Published
2015-11-23
Volume
17
Issue
1
Pages
19-32
ISSN
  • 1471-0056
  • 1471-0064
DOI
10.1038/nrg.2015.3
PMC
PMC5993438
Cited by
1,163
References
25
Language
en
Licence
www.springer.com/tdm (tdm)

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