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Items: 1 to 20 of 286

1.

Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification.

Evers MM, Tran HD, Zalachoras I, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM.

Nucleic Acid Ther. 2014 Feb;24(1):4-12. doi: 10.1089/nat.2013.0452. Epub 2013 Dec 31.

PMID:
24380395
2.

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.

Carroll JB, Warby SC, Southwell AL, Doty CN, Greenlee S, Skotte N, Hung G, Bennett CF, Freier SM, Hayden MR.

Mol Ther. 2011 Dec;19(12):2178-85. doi: 10.1038/mt.2011.201. Epub 2011 Oct 4.

3.

Wild type Huntingtin reduces the cellular toxicity of mutant Huntingtin in mammalian cell models of Huntington's disease.

Ho LW, Brown R, Maxwell M, Wyttenbach A, Rubinsztein DC.

J Med Genet. 2001 Jul;38(7):450-2.

4.

Caspase-6 activity in a BACHD mouse modulates steady-state levels of mutant huntingtin protein but is not necessary for production of a 586 amino acid proteolytic fragment.

Gafni J, Papanikolaou T, Degiacomo F, Holcomb J, Chen S, Menalled L, Kudwa A, Fitzpatrick J, Miller S, Ramboz S, Tuunanen PI, Lehtimäki KK, Yang XW, Park L, Kwak S, Howland D, Park H, Ellerby LM.

J Neurosci. 2012 May 30;32(22):7454-65. doi: 10.1523/JNEUROSCI.6379-11.2012.

5.

Transgenic mouse model expressing the caspase 6 fragment of mutant huntingtin.

Waldron-Roby E, Ratovitski T, Wang X, Jiang M, Watkin E, Arbez N, Graham RK, Hayden MR, Hou Z, Mori S, Swing D, Pletnikov M, Duan W, Tessarollo L, Ross CA.

J Neurosci. 2012 Jan 4;32(1):183-93. doi: 10.1523/JNEUROSCI.1305-11.2012.

6.

In vivo proof-of-concept of removal of the huntingtin caspase cleavage motif-encoding exon 12 approach in the YAC128 mouse model of Huntington's disease.

Casaca-Carreira J, Toonen LJA, Evers MM, Jahanshahi A, van-Roon-Mom WMC, Temel Y.

Biomed Pharmacother. 2016 Dec;84:93-96. doi: 10.1016/j.biopha.2016.09.007. Epub 2016 Sep 16.

PMID:
27639545
8.

N-terminal mutant huntingtin associates with mitochondria and impairs mitochondrial trafficking.

Orr AL, Li S, Wang CE, Li H, Wang J, Rong J, Xu X, Mastroberardino PG, Greenamyre JT, Li XJ.

J Neurosci. 2008 Mar 12;28(11):2783-92. doi: 10.1523/JNEUROSCI.0106-08.2008.

9.

Caspase cleavage of mutant huntingtin precedes neurodegeneration in Huntington's disease.

Wellington CL, Ellerby LM, Gutekunst CA, Rogers D, Warby S, Graham RK, Loubser O, van Raamsdonk J, Singaraja R, Yang YZ, Gafni J, Bredesen D, Hersch SM, Leavitt BR, Roy S, Nicholson DW, Hayden MR.

J Neurosci. 2002 Sep 15;22(18):7862-72.

10.

Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease.

Sathasivam K, Neueder A, Gipson TA, Landles C, Benjamin AC, Bondulich MK, Smith DL, Faull RL, Roos RA, Howland D, Detloff PJ, Housman DE, Bates GP.

Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):2366-70. doi: 10.1073/pnas.1221891110. Epub 2013 Jan 22.

11.
12.

Mutant huntingtin causes context-dependent neurodegeneration in mice with Huntington's disease.

Yu ZX, Li SH, Evans J, Pillarisetti A, Li H, Li XJ.

J Neurosci. 2003 Mar 15;23(6):2193-202.

13.

Full length mutant huntingtin is required for altered Ca2+ signaling and apoptosis of striatal neurons in the YAC mouse model of Huntington's disease.

Zhang H, Li Q, Graham RK, Slow E, Hayden MR, Bezprozvanny I.

Neurobiol Dis. 2008 Jul;31(1):80-8. doi: 10.1016/j.nbd.2008.03.010. Epub 2008 Apr 16.

14.

Transgenic mice expressing caspase-6-derived N-terminal fragments of mutant huntingtin develop neurologic abnormalities with predominant cytoplasmic inclusion pathology composed largely of a smaller proteolytic derivative.

Tebbenkamp AT, Green C, Xu G, Denovan-Wright EM, Rising AC, Fromholt SE, Brown HH, Swing D, Mandel RJ, Tessarollo L, Borchelt DR.

Hum Mol Genet. 2011 Jul 15;20(14):2770-82. doi: 10.1093/hmg/ddr176. Epub 2011 Apr 22.

16.

Amyloid formation by mutant huntingtin: threshold, progressivity and recruitment of normal polyglutamine proteins.

Huang CC, Faber PW, Persichetti F, Mittal V, Vonsattel JP, MacDonald ME, Gusella JF.

Somat Cell Mol Genet. 1998 Jul;24(4):217-33.

PMID:
10410676
17.

Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.

Gagnon KT, Pendergraff HM, Deleavey GF, Swayze EE, Potier P, Randolph J, Roesch EB, Chattopadhyaya J, Damha MJ, Bennett CF, Montaillier C, Lemaitre M, Corey DR.

Biochemistry. 2010 Nov 30;49(47):10166-78. doi: 10.1021/bi101208k. Epub 2010 Nov 8.

18.

Comparison of huntingtin proteolytic fragments in human lymphoblast cell lines and human brain.

Toneff T, Mende-Mueller L, Wu Y, Hwang SR, Bundey R, Thompson LM, Chesselet MF, Hook V.

J Neurochem. 2002 Jul;82(1):84-92.

19.

Targeting several CAG expansion diseases by a single antisense oligonucleotide.

Evers MM, Pepers BA, van Deutekom JC, Mulders SA, den Dunnen JT, Aartsma-Rus A, van Ommen GJ, van Roon-Mom WM.

PLoS One. 2011;6(9):e24308. doi: 10.1371/journal.pone.0024308. Epub 2011 Sep 1.

20.

Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.

Sun X, Marque LO, Cordner Z, Pruitt JL, Bhat M, Li PP, Kannan G, Ladenheim EE, Moran TH, Margolis RL, Rudnicki DD.

Hum Mol Genet. 2014 Dec 1;23(23):6302-17. doi: 10.1093/hmg/ddu349. Epub 2014 Jul 4.

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