TabriziSJ, GhoshR, LeavittBR. Huntingtin lowering strategies for disease modification in Huntington's disease. Neuron, 2019; 101:801–819.
3.
KwonD. Failure of genetic therapies for Huntington's devastates community. Nature, 2021; 593:180.
4.
HsuPD, LanderES, ZhangF. Development and applications of CRISPR-Cas9 for genome engineering. Cell, 2014; 157:1262–1278.
5.
GillmoreJD, GaneE, TaubelJ, et al.CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med, 2021; 385:493–502.
6.
PfisterEL, KenningtonL, StraubhaarJ, et al.Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr Biol, 2009; 19:774–778.
7.
OikemusSR, PfisterE, SappE, et al.Allele-specific knockdown of mutant huntingtin protein via editing at coding region single nucleotide polymorphism heterozygosities. Hum Gene Ther, 2021; 33:25–36.
8.
YangS, ChangR, YangH, et al.CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease. J Clin Invest, 2017; 127:2719–2724.
9.
EkmanFK, OjalaDS, AdilMM, et al.CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington's Disease Mouse Model. Mol Ther Nucleic Acids, 2019; 17:829–839.