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

[41] Accumulating gene errors drive Huntington's disease
Rajagopal, Veera M.
Gelfman, Sahar
NATURE, 2025, 639 (8055) : 584 - 586
[42] Gene Expression Profiling of Depression in Huntington's Disease
Colpo, Gabriela D.
Rocha, Natalia P.
Stimming, Erin Furr
Teixeira, Antonio L.
NEUROTHERAPEUTICS, 2019, 16 (04) : 1355 - 1355
[43] Moving toward a gene therapy for Huntington’s disease
J C Glorioso
J B Cohen
D L Carlisle
I Munoz-Sanjuan
R M Friedlander
Gene Therapy, 2015, 22 : 931 - 933
[44] Prospectives for cell and gene therapy in Huntington's disease
Bachoud-Levi, AC
Hantraye, P
Peschanski, M
NEURONAL DEGENERATION AND REGENERATION: FROM BASIC MECHANISMS TO PROSPECTS FOR THERAPY, 1998, 117 : 511 - 524
[45] An update on Huntington's disease: from the gene to the clinic
Kim, Samuel D.
Fung, Victor S. C.
CURRENT OPINION IN NEUROLOGY, 2014, 27 (04) : 477 - 483
[46] Revisiting cell and gene therapies in Huntington's disease
Beatriz, Margarida
Lopes, Carla
Ribeiro, Ana Claudia S.
Rego, Ana Cristina Carvalho
JOURNAL OF NEUROSCIENCE RESEARCH, 2021, 99 (07) : 1744 - 1762
[47] Chromosomal profiles of gene expression in Huntington's disease
Anderson, Alexander N.
Roncaroli, Federico
Hodges, Angela
Deprez, Manuel
Turkheimer, Federico E.
BRAIN, 2008, 131 : 381 - 388
[48] Gene Expression Profiling of depression in Huntington's disease
Colpo, G.
Rocha, N.
Stimming, E. Furr
Lucio, A.
MOVEMENT DISORDERS, 2019, 34 : S4 - S4
[49] Moving toward a gene therapy for Huntington's disease
Glorioso, J. C.
Cohen, J. B.
Carlisle, D. L.
Munoz-Sanjuan, I.
Friedlander, R. M.
GENE THERAPY, 2015, 22 (12) : 931 - 933
[50] Gene silencing approaches for the treatment of Huntington's disease
Merienne, Nicolas
Deglon, Nicole
M S-MEDECINE SCIENCES, 2015, 31 (02): : 159 - 167

← 1 2 3 4 5 →