4.4 Article

RNA-Guided Adenosine Deaminases: Advances and Challenges for Therapeutic RNA Editing

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BIOCHEMISTRY
卷 58, 期 15, 页码 1947-1957

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AMER CHEMICAL SOC
DOI: 10.1021/acs.biochem.9b00046

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资金

  1. Burroughs Wellcome Fund [1013926]
  2. National Institutes of Health [R01HG009285, RO1CA222826, RO1GM123313]

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Targeted transcriptome engineering, in contrast to genome engineering, offers a complementary and potentially tunable and reversible strategy for cellular engineering. In this regard, adenosine to inosine (A-to-I) RNA base editing was recently engineered to make programmable base conversions on target RNAs. Similar to the DNA base editing technology, A-to-I RNA editing may offer an attractive alternative in a therapeutic setting, especially for the correction of point mutations. This Perspective introduces five currently characterized RNA editing systems and serves as a reader's guide for implementing an appropriate RNA editing strategy for applications in research or therapeutics.

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