Hunting out the repeat expansion in Huntington’s pigs

Guang Yang, Boxun Lu

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Protein Cell ›› 2023, Vol. 14 ›› Issue (12) : 871-873. DOI: 10.1093/procel/pwad014
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Hunting out the repeat expansion in Huntington’s pigs

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Guang Yang, Boxun Lu. Hunting out the repeat expansion in Huntington’s pigs. Protein Cell, 2023, 14(12): 871‒873 https://doi.org/10.1093/procel/pwad014

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.
CrossRef Google scholar
[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.
CrossRef Google scholar
[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.
CrossRef Google scholar
[4]
Hsu, PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell 2014;157:1262–1278.
CrossRef Google scholar
[5]
Ledford H. CRISPR treatment inserted directly into the body for first time. Nature 2020;579:185.
CrossRef Google scholar
[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.
CrossRef Google scholar
[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.
CrossRef Google scholar
[8]
Rees HA, Liu DR. Base editing: precision chemistry on the genome and transcriptome of living cells. Nat Rev Genet 2018;19:770–788.
CrossRef Google scholar
[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.
CrossRef Google scholar
[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.
CrossRef Google scholar
[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.
CrossRef Google scholar
[12]
Zeng J, Wu Y, Ren C et al. Therapeutic base editing of human hematopoietic stem cells. Nat Med 2020;26:535–541.
CrossRef Google scholar

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