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Comment
. 2021 Sep;18(9):597-598.
doi: 10.1038/s41575-021-00491-9.

Therapeutic base editing in the adult liver

Coen C Paulusma  1 , Piter J Bosma  2
Affiliations
Comment

Therapeutic base editing in the adult liver

Coen C Paulusma et al. Nat Rev Gastroenterol Hepatol. 2021 Sep.
No abstract available

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Comment on

  • In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.
    Musunuru K, Chadwick AC, Mizoguchi T, Garcia SP, DeNizio JE, Reiss CW, Wang K, Iyer S, Dutta C, Clendaniel V, Amaonye M, Beach A, Berth K, Biswas S, Braun MC, Chen HM, Colace TV, Ganey JD, Gangopadhyay SA, Garrity R, Kasiewicz LN, Lavoie J, Madsen JA, Matsumoto Y, Mazzola AM, Nasrullah YS, Nneji J, Ren H, Sanjeev A, Shay M, Stahley MR, Fan SHY, Tam YK, Gaudelli NM, Ciaramella G, Stolz LE, Malyala P, Cheng CJ, Rajeev KG, Rohde E, Bellinger AM, Kathiresan S. Musunuru K, et al. Nature. 2021 May;593(7859):429-434. doi: 10.1038/s41586-021-03534-y. Epub 2021 May 19. Nature. 2021. PMID: 34012082
  • In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
    Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, Qi W, Hruzova M, Kissling L, Lenggenhager D, Borrelli C, Egli S, Frey N, Bakker N, Walker JA 2nd, Kadina AP, Victorov DV, Pacesa M, Kreutzer S, Kontarakis Z, Moor A, Jinek M, Weissman D, Stoffel M, van Boxtel R, Holden K, Pardi N, Thöny B, Häberle J, Tam YK, Semple SC, Schwank G. Rothgangl T, et al. Nat Biotechnol. 2021 Aug;39(8):949-957. doi: 10.1038/s41587-021-00933-4. Epub 2021 May 19. Nat Biotechnol. 2021. PMID: 34012094 Free PMC article.

References

    1. Anzalone, A. V. et al. Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Nat. Biotechnol. 38, 824–844 (2020). - DOI
    1. Rothgangl, T. et al. In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nat. Biotechnol. https://doi.org/10.1038/s41587-021-00933-4 (2021). - DOI - PubMed
    1. Musunuru, K. et al. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature 593, 429–434 (2021). - DOI
    1. Song, C.-Q. et al. Adenine base editing in an adult mouse model of tyrosinaemia. Nat. Biomed. Eng. 4 (2020).
    1. Villiger, L. et al. Treatment of a metabolic liver disease by in vivo genome base editing in adult mice. Nat. Med. 24, 1519–1525 (2018). - DOI

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