Gene targeting techniques for Huntington's disease

被引：18

作者：

Fields, Eric ^{[1
]}

Vaughan, Erik ^{[1
]}

Tripu, Deepika ^{[1
]}

Lim, Isabelle ^{[1
]}

Shrout, Katherine ^{[1
]}

Conway, Jessica ^{[1
]}

Salib, Nicole ^{[1
]}

Lee, Yubin ^{[1
]}

Dhamsania, Akash ^{[1
]}

Jacobsen, Michael ^{[1
]}

Woo, Ashley ^{[1
]}

Xue, Huijing ^{[2
]}

Cao, Kan ^{[1
,2
]}

机构：

[1] Univ Maryland, Gemstone Honors Program, College Pk, MD 20742 USA

[2] Univ Maryland, Dept Cell Biol & Mol Genet, College Pk, MD 20742 USA

来源：

AGEING RESEARCH REVIEWS | 2021年 / 70卷

关键词：

Huntington's disease; Allele-specific targeting; Prime editing; SNPs; MUTANT HUNTINGTIN; CAG REPEAT; GUT MICROBIOTA; MOUSE MODEL; RNA; MECHANISMS; THERAPY; PHOSPHORYLATION; INACTIVATION; CILIOGENESIS;

D O I：

10.1016/j.arr.2021.101385

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Huntington's disease (HD) is an autosomal neurodegenerative disorder caused by extended trinucleotide CAG repetition in the HTT gene. Wild-type huntingtin protein (HTT) is essential, involved in a variety of crucial cellular functions such as vesicle transportation, cell division, transcription regulation, autophagy, and tissue maintenance. The mutant HTT (mHTT) proteins in the body interfere with HTT's normal cellular functions and cause additional detrimental effects. In this review, we discuss multiple approaches targeting DNA and RNA to reduce mHTT expression. These approaches are categorized into non-allele-specific silencing and allele-specificsilencing using Single Nucleotide Polymorphisms (SNPs) and haplogroup analysis. Additionally, this review discusses a potential application of recent CRISPR prime editing technology in targeting HD.

引用

页数：8

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