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Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases.

Author information

Affiliations

  • 1. Department of Pharmacology, Yonsei University College of Medicine, Seoul, Republic of Korea.
      Authors
    • Jang H 1, 6
    • Shin JH 1, 6
    • Yu G 1, 6
    • Gopalappa R 1
    • Kim HH 1, 6
    (5 authors)
  • 2. Department of Anatomy and Cell Biology, Seoul National University College of Medicine, Seoul, Republic of Korea.
      Authors
    • Jo DH 2
    (1 author)
  • 3. Fight against Angiogenesis-Related Blindness (FARB) Laboratory, Biomedical Research Institute, Seoul National University Hospital, Seoul, Republic of Korea.
      Authors
    • Cho CS 3
    • Kim JH 3
    (2 authors)
  • 4. Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, Republic of Korea.
      Authors
    • Seo JH 4
    • Cho SR 4, 6
    (2 authors)
  • 5. Genome Editing Research Center, Korea Research Institute of Bioscience and Biotechnology (KRIBB), Daejeon, Republic of Korea.
      Authors
    • Kim D 5
    (1 author)
    • Show all (6)

ORCIDs linked to this article

Nature Biomedical Engineering, 26 Aug 2021, 6(2):181-194
https://doi.org/10.1038/s41551-021-00788-9 PMID: 34446856 

Abstract 

The use of prime editing-a gene-editing technique that induces small genetic changes without the need for donor DNA and without causing double strand breaks-to correct pathogenic mutations and phenotypes needs to be tested in animal models of human genetic diseases. Here we report the use of prime editors 2 and 3, delivered by hydrodynamic injection, in mice with the genetic liver disease hereditary tyrosinemia, and of prime editor 2, delivered by an adeno-associated virus vector, in mice with the genetic eye disease Leber congenital amaurosis. For each pathogenic mutation, we identified an optimal prime-editing guide RNA by using cells transduced with lentiviral libraries of guide-RNA-encoding sequences paired with the corresponding target sequences. The prime editors precisely corrected the disease-causing mutations and led to the amelioration of the disease phenotypes in the mice, without detectable off-target edits. Prime editing should be tested further in more animal models of genetic diseases.

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Read article at publisher's site: https://doi.org/10.1038/s41551-021-00788-9

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Funding 

Funders who supported this work.

National Research Foundation of Korea (6)

the Korea Research Institute of Bioscience and Biotechnology(KRIBB) Research Initiative Program

    the New Faculty Startup Fund from Seoul National University