Hunting out the repeat expansion in Huntington’s pigs

References

[1] Anzalone AV, Randolph PB, Davis JR et al. Search-and-replace genome editing without double-strand breaks or donor DNA. Nature 2019;576:149–157.

[2] Enache OM, Rendo V, Abdusamad M et al. Cas9 activates the p53 pathway and selects for p53-inactivating mutations. Nat Genet 2020;52:662–668.

[3] Gardiner SL, Boogaard MW, Trompet S et al. Prevalence of carriers of intermediate and pathological polyglutamine disease-associated alleles among large population-based cohorts. JAMA Neurol 2019;76:650–656.

[4] Hsu, PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell 2014;157:1262–1278.

[5] Ledford H. CRISPR treatment inserted directly into the body for first time. Nature 2020;579:185.

[6] Li Z, Wang C, Wang Z et al. Allele-selective lowering of mutant HTT protein by HTT-LC3 linker compounds. Nature 2019;575:203–209.

[7] Macdonald M. A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington’s disease chromosomes. Cell 1993;72:971–983.

[8] Rees HA, Liu DR. Base editing: precision chemistry on the genome and transcriptome of living cells. Nat Rev Genet 2018;19:770–788.

[9] Tabrizi SJ, Leavitt BR, Landwehrmeyer GB et al.; Phase 1–2a IONIS-HTTRx Study Site Teams. Targeting Huntingtin expression in patients with Huntington’s disease. N Engl J Med 2019;380:2307–2316.

[10] Yan S, Tu Z, Liu Z et al. A Huntingtin Knockin Pig model recapitulates features of selective neurodegeneration in Huntington’s disease. Cell 2018;173, 989–1002.e1013.

[11] Yan S, Zheng X, Lin Y et al. Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease. Nat Biomed Eng 2023:1–18.

[12] Zeng J, Wu Y, Ren C et al. Therapeutic base editing of human hematopoietic stem cells. Nat Med 2020;26:535–541.

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The Author(s) 2023. Published by Oxford University Press on behalf of Higher Education Press.