Gene targeting techniques for Huntington's disease.

Szczegóły
Abstrakt

Tytuł:: Gene targeting techniques for Huntington's disease.
Autorzy:: Fields E; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Vaughan E; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Tripu D; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Lim I; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Shrout K; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Conway J; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Salib N; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Lee Y; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Dhamsania A; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Jacobsen M; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Woo A; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States.
Xue H; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20742, United States.
Cao K; Gemstone Honors Program, University of Maryland, College Park, MD 20742, United States; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20742, United States. Electronic address: .
Źródło:: Ageing research reviews [Ageing Res Rev] 2021 Sep; Vol. 70, pp. 101385. Date of Electronic Publication: 2021 Jun 05.
Typ publikacji:: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review
Język:: English
Imprint Name(s):: Original Publication: Oxford, UK : Elsevier Science, c2002-
MeSH Terms:: Huntington Disease*/genetics
Huntington Disease*/therapy
Alleles ; Gene Expression Regulation ; Gene Targeting ; Humans ; Huntingtin Protein/genetics ; Huntingtin Protein/metabolism
References:: J Cell Biol. 2009 Dec 28;187(7):1083-99. (PMID: 20026656)
Sci Rep. 2018 May 25;8(1):8096. (PMID: 29802276)
Nature. 2019 Dec;576(7785):149-157. (PMID: 31634902)
J Huntingtons Dis. 2021;10(1):203-220. (PMID: 32925081)
J Huntingtons Dis. 2020;9(2):115-128. (PMID: 32417788)
Neuron. 2015 Nov 18;88(4):667-77. (PMID: 26590344)
Alzheimers Dement (N Y). 2018 Dec 17;4:746-755. (PMID: 30581980)
Acta Pharmacol Sin. 2018 May;39(5):754-769. (PMID: 29620053)
Sci Rep. 2017 Oct 19;7(1):13537. (PMID: 29051531)
Nat Rev Mol Cell Biol. 2008 Jan;9(1):22-32. (PMID: 18073770)
Gene. 1992 Nov 16;121(2):189-94. (PMID: 1332911)
Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):2366-70. (PMID: 23341618)
J Neurol Neurosurg Psychiatry. 2001 Sep;71(3):310-4. (PMID: 11511702)
Hum Mol Genet. 1999 Mar;8(3):397-407. (PMID: 9949199)
Brain. 2012 Apr;135(Pt 4):1197-209. (PMID: 22252996)
Nat Rev Neurol. 2020 Oct;16(10):529-546. (PMID: 32796930)
Proc Natl Acad Sci U S A. 2012 Jan 10;109(2):594-9. (PMID: 22184244)
Cell. 2012 Aug 31;150(5):895-908. (PMID: 22939619)
PLoS One. 2013 Sep 03;8(9):e73902. (PMID: 24019939)
J Neurol Sci. 2009 Jan 15;276(1-2):159-62. (PMID: 18977004)
Lancet Neurol. 2019 Jun;18(6):549-558. (PMID: 31122495)
J Neurosci. 2009 Oct 28;29(43):13589-602. (PMID: 19864571)
Nature. 2006 Dec 21;444(7122):1027-31. (PMID: 17183312)
Cell. 1998 Oct 2;95(1):55-66. (PMID: 9778247)
Trends Neurosci. 2013 May;36(5):305-12. (PMID: 23384445)
J Huntingtons Dis. 2018;7(1):89-98. (PMID: 29480210)
Cell. 1995 Jun 2;81(5):811-23. (PMID: 7774020)
EBioMedicine. 2017 Oct;24:166-178. (PMID: 28965876)
Neurology. 2012 Mar 6;78(10):690-5. (PMID: 22323755)
Eur J Hum Genet. 2017 Nov;25(11):1202-1209. (PMID: 28832564)
Neurol Genet. 2019 Apr 01;5(2):e323. (PMID: 31119194)
Stem Cell Reports. 2014 Apr 03;2(4):491-506. (PMID: 24749073)
Front Pharmacol. 2019 Mar 29;10:305. (PMID: 30983999)
Neuron. 2012 Jun 21;74(6):964-6. (PMID: 22726826)
PLoS One. 2017 Feb 9;12(2):e0171127. (PMID: 28182673)
J Neurosci. 2002 May 1;22(9):3473-83. (PMID: 11978824)
J Cell Biol. 2005 May 23;169(4):647-56. (PMID: 15911879)
Mol Ther. 2011 Dec;19(12):2152-62. (PMID: 22031240)
Mol Ther. 2009 Jun;17(6):1053-63. (PMID: 19240687)
Cell. 1996 Nov 1;87(3):493-506. (PMID: 8898202)
Ann Neurol. 2009 Mar;65(3):276-85. (PMID: 19334076)
Hum Mol Genet. 2016 Oct 15;25(20):4566-4576. (PMID: 28172889)
Mol Ther. 2017 Jan 4;25(1):12-23. (PMID: 28129107)
Nat Biotechnol. 2020 Jul;38(7):824-844. (PMID: 32572269)
Nature. 2019 Nov;575(7781):203-209. (PMID: 31666698)
Neurobiol Dis. 2020 Nov;145:105076. (PMID: 32898646)
Curr Trends Neurol. 2011 Jan 1;5:65-78. (PMID: 22180703)
N Engl J Med. 2019 Jun 13;380(24):2307-2316. (PMID: 31059641)
JAMA Neurol. 2020 Jun 1;77(6):764-772. (PMID: 32202594)
Neurobiol Dis. 2020 Aug;142:104958. (PMID: 32526274)
Sci Rep. 2020 Oct 26;10(1):18270. (PMID: 33106549)
Neuron. 2012 Jun 21;74(6):1031-44. (PMID: 22726834)
J Huntingtons Dis. 2020;9(3):217-229. (PMID: 32925079)
Front Cell Neurosci. 2020 Jan 17;13:574. (PMID: 32009905)
Hum Mol Genet. 2002 Nov 1;11(23):2905-17. (PMID: 12393802)
Neurobiol Dis. 2020 Dec;146:105120. (PMID: 32991997)
Nature. 2012 Oct 4;490(7418):55-60. (PMID: 23023125)
Dev Cell. 2002 Jun;2(6):831-7. (PMID: 12062094)
Neurobiol Dis. 2020 Feb;135:104268. (PMID: 30194046)
EMBO Rep. 2004 Oct;5(10):958-63. (PMID: 15459747)
PLoS Genet. 2018 Apr 5;14(4):e1007308. (PMID: 29621242)
Nature. 2021 Jan;589(7843):608-614. (PMID: 33408413)
J Clin Invest. 2016 Nov 1;126(11):4319-4330. (PMID: 27721240)
Neuron. 2005 Jul 7;47(1):29-41. (PMID: 15996546)
Neurol Int. 2018 Jul 04;10(2):7473. (PMID: 30069288)
Annu Rev Pharmacol Toxicol. 2010;50:259-93. (PMID: 20055705)
Neuron. 2017 Apr 5;94(1):93-107.e6. (PMID: 28384479)
Nat Med. 2014 May;20(5):536-41. (PMID: 24784230)
Hum Gene Ther. 2014 May;25(5):461-74. (PMID: 24484067)
Mov Disord. 2015 Mar;30(3):350-8. (PMID: 25476529)
Brain Commun. 2020 Jul 24;2(2):fcaa110. (PMID: 33005892)
Nat Genet. 2000 Nov;26(3):300-6. (PMID: 11062468)
Nat Biotechnol. 2012 Nov;30(11):1063. (PMID: 23138301)
J Huntingtons Dis. 2018;7(4):289-296. (PMID: 30452421)
Lancet. 2007 Jan 20;369(9557):218-28. (PMID: 17240289)
Nat Protoc. 2013 Nov;8(11):2281-2308. (PMID: 24157548)
Cell Transplant. 2014;23(4-5):441-58. (PMID: 24816443)
Mol Cell Biol. 2009 Nov;29(22):6106-16. (PMID: 19752198)
Dev Biol. 2015 Dec 15;408(2):305-15. (PMID: 26192473)
Proc Natl Acad Sci U S A. 2016 Mar 22;113(12):3359-64. (PMID: 26951659)
Neurology. 2008 Nov 4;71(19):1506-13. (PMID: 18981372)
Nat Rev Dis Primers. 2015 Apr 23;1:15005. (PMID: 27188817)
Int Rev Neurobiol. 2011;98:373-418. (PMID: 21907095)
J Clin Invest. 2011 Nov;121(11):4372-82. (PMID: 21985783)
Science. 2020 Aug 14;369(6505):787-793. (PMID: 32675289)
Neuroscientist. 2011 Oct;17(5):475-92. (PMID: 21311053)
Lancet Neurol. 2017 Oct;16(10):837-847. (PMID: 28920889)
Grant Information:: R01 HL126784 United States HL NHLBI NIH HHS
Contributed Indexing:: Keywords: Allele-specific targeting; Huntington’s disease; Prime editing; SNPs
Substance Nomenclature:: 0 (Huntingtin Protein)
Entry Date(s):: Date Created: 20210607 Date Completed: 20210910 Latest Revision: 20231107
Update Code:: 20240104
PubMed Central ID:: PMC8373677
DOI:: 10.1016/j.arr.2021.101385
PMID:: 34098113
: Czasopismo naukowe

Full Text Finder

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-specific-silencing using Single Nucleotide Polymorphisms (SNPs) and haplogroup analysis. Additionally, this review discusses a potential application of recent CRISPR prime editing technology in targeting HD.
(Copyright © 2021 The Author(s). Published by Elsevier B.V. All rights reserved.)

Informacja