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

[1] Targeting mitochondria in Huntington's disease
Oliveira, Jorge
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION, 2019, 49 : 21 - 22
[2] Selection of an AAV gene therapy targeting huntingtin for the treatment of Huntington's disease
Sah, D.
Zhou, P.
Chen, F.
Wang, X.
Christensen, E.
Thompson, J.
Nonnenmacher, M.
Scheel, M.
Ren, X.
Wang, W.
Zhou, X.
Stanek, L.
Mastis, B.
Pechan, P.
Horowitz, E.
Dismuke, D.
Kells, A.
Carter, T.
Hou, J.
HUMAN GENE THERAPY, 2017, 28 (12) : A74 - A75
[3] Targeting the proteostasis network in Huntington's disease
Soares, Tania R.
Reis, Sara D.
Pinho, Brigida R.
Duchen, Michael R.
Oliveira, Jorge M. A.
AGEING RESEARCH REVIEWS, 2019, 49 : 92 - 103
[4] Targeting energy metabolism in Huntington's disease
Walker, FO
Raymond, LA
LANCET, 2004, 364 (9431): : 312 - 313
[5] Huntington's disease: From disease to gene
Tamminga, CA
Schwarcz, R
MacDonald, ME
AMERICAN JOURNAL OF PSYCHIATRY, 1997, 154 (06): : 725 - 725
[6] Gene silencing in Huntington's disease
Alison Rowan
Nature Reviews Neuroscience, 2005, 6 : 422 - 422
[7] Gene therapy for Huntington's disease
Ramaswamy, Shilpa
Kordower, Jeffrey H.
NEUROBIOLOGY OF DISEASE, 2012, 48 (02) : 243 - 254
[8] The Huntington's Disease Gene Discovery
Franklin, Gustavo L.
Teive, Helio A. G.
Tensini, Fernando Spina
Camargo, Carlos Henrique Ferreira
de Lima, Nayra de Souza Carvalho
de dos Santos, Diego de Castro
Meira, Alex T.
Tabrizi, Sarah J.
MOVEMENT DISORDERS, 2024, 39 (02) : 227 - 234
[9] New techniques for understanding Huntington's disease
Hurley, RA
Jackson, EF
Fisher, RE
Taber, KH
JOURNAL OF NEUROPSYCHIATRY AND CLINICAL NEUROSCIENCES, 1999, 11 (02) : 173 - 175
[10] Targeting Huntington Disease
不详
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2012, 308 (05): : 444 - 444

← 1 2 3 4 5 →