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

共 50 条

[31] Gene Therapy for Huntington's Disease: The Future Is in Gene Editing
Batista, Ana Rita
HUMAN GENE THERAPY, 2022, 33 (1-2) : 12 - 13
[32] SREBP2 gene therapy targeting striatal astrocytes ameliorates Huntington's disease phenotypes
Birolini, Giulia
Verlengia, Gianluca
Talpo, Francesca
Maniezzi, Claudia
Zentilin, Lorena
Giacca, Mauro
Conforti, Paola
Cordiglieri, Chiara
Caccia, Claudio
Leoni, Valerio
Taroni, Franco
Biella, Gerardo
Simonato, Michele
Cattaneo, Elena
Valenza, Marta
BRAIN, 2021, 144 : 3175 - 3190
[33] Development of an AAV-Based, RNA-Targeting Gene Therapy for the Treatment of Huntington's Disease
Geddes, Claire E.
Roth, Daniela
Ta, Angeline
Wilson, Alistair
Gutierrez, Haydee
Narayan, Nandini
Angelis, Dimitrios
Fang, Lydia
Berlin, Aaron
Nachtrab, Greg
Gibbs, Dan
Zisoulis, Dimitrios
Batra, Ranjan
MOLECULAR THERAPY, 2021, 29 (04) : 271 - 271
[34] Targeting Glial Cells to Elucidate the Pathogenesis of Huntington’s Disease
Han-Yun Hsiao
Yijuang Chern
Molecular Neurobiology, 2010, 41 : 248 - 255
[35] New therapies in Huntington's disease by directly targeting the brain
Rosser, A.
EUROPEAN NEUROPSYCHOPHARMACOLOGY, 2019, 29 : S34 - S34
[36] Targeting Tau to Treat Clinical Features of Huntington's Disease
Masnata, Maria
Salem, Shireen
Jacquet, Aurelie de Rus
Anwer, Mehwish
Cicchetti, Francesca
FRONTIERS IN NEUROLOGY, 2020, 11
[37] Therapeutic targeting of Huntington's disease: Molecular and clinical approaches
Kumar, Dhiraj
Hasan, Gulam Mustafa
Islam, Asimul
Hassan, Md. Imtaiyaz
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2023, 655 : 18 - 24
[38] Intervention in Huntington's disease: targeting serotonin and BDNF signaling
Duan, W
Mattson, MP
JOURNAL OF NEUROCHEMISTRY, 2004, 90 : 110 - 110
[39] Targeting Glial Cells to Elucidate the Pathogenesis of Huntington's Disease
Hsiao, Han-Yun
Chern, Yijuang
MOLECULAR NEUROBIOLOGY, 2010, 41 (2-3) : 248 - 255
[40] Oxidative stress in Huntington's disease patients, asymptomatic Huntington's disease gene carriers and healthy subjects
Klepac, N.
Relja, M.
EUROPEAN JOURNAL OF NEUROLOGY, 2007, 14 : 11 - 11

← 1 2 3 4 5 →