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Literature DB >> 22726826

"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease.

Abstract

Lowering mutant Huntingtin is a consensus therapeutic strategy for Huntington's disease. In this issue of Neuron, Kordasiewicz et al. (2012) show the benefit of transient antisense oligonucleotide (ASO) therapy to degrade Huntingtin mRNA and elicit sustained therapeutic benefit in HD mice.

Entities: Chemical Disease Gene Species

Mesh：

Substances：

Year: 2012 PMID： 22726826 PMCID： PMC3513277 DOI： 10.1016/j.neuron.2012.06.001

Source DB: PubMed Journal: Neuron ISSN： 0896-6273 Impact factor: 17.173

14 in total

1. Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease.

Authors: A Yamamoto; J J Lucas; R Hen
Journal: Cell Date: 2000-03-31 Impact factor: 41.582

2. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

Authors: Holly B Kordasiewicz; Lisa M Stanek; Edward V Wancewicz; Curt Mazur; Melissa M McAlonis; Kimberly A Pytel; Jonathan W Artates; Andreas Weiss; Seng H Cheng; Lamya S Shihabuddin; Gene Hung; C Frank Bennett; Don W Cleveland
Journal: Neuron Date: 2012-06-21 Impact factor: 17.173

3. Pathological cell-cell interactions elicited by a neuropathogenic form of mutant Huntingtin contribute to cortical pathogenesis in HD mice.

Authors: Xiaofeng Gu; Chenjian Li; Weizheng Wei; Victor Lo; Shiaoching Gong; Shi-Hua Li; Takuji Iwasato; Shigeyoshi Itohara; Xiao-Jiang Li; Istvan Mody; Nathaniel Heintz; X William Yang
Journal: Neuron Date: 2005-05-05 Impact factor: 17.173

4. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.

Authors: Scott Q Harper; Patrick D Staber; Xiaohua He; Steven L Eliason; Inês H Martins; Qinwen Mao; Linda Yang; Robert M Kotin; Henry L Paulson; Beverly L Davidson
Journal: Proc Natl Acad Sci U S A Date: 2005-04-05 Impact factor: 11.205

5. Antisense oligonucleotide therapy for neurodegenerative disease.

Authors: Richard A Smith; Timothy M Miller; Koji Yamanaka; Brett P Monia; Thomas P Condon; Gene Hung; Christian S Lobsiger; Chris M Ward; Melissa McAlonis-Downes; Hongbing Wei; Ed V Wancewicz; C Frank Bennett; Don W Cleveland
Journal: J Clin Invest Date: 2006-07-27 Impact factor: 14.808

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

Authors: Jeffrey B Carroll; Simon C Warby; Amber L Southwell; Crystal N Doty; Sarah Greenlee; Niels Skotte; Gene Hung; C Frank Bennett; Susan M Freier; Michael R Hayden
Journal: Mol Ther Date: 2011-10-04 Impact factor: 11.454

7. Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice.

Authors: I Dragatsis; M S Levine; S Zeitlin
Journal: Nat Genet Date: 2000-11 Impact factor: 38.330

8. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.

Authors: M DiFiglia; M Sena-Esteves; K Chase; E Sapp; E Pfister; M Sass; J Yoder; P Reeves; R K Pandey; K G Rajeev; M Manoharan; D W Y Sah; P D Zamore; N Aronin
Journal: Proc Natl Acad Sci U S A Date: 2007-10-16 Impact factor: 11.205

9. Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease.

Authors: Elizabeth J Slow; Jeremy van Raamsdonk; Daniel Rogers; Sarah H Coleman; Rona K Graham; Yu Deng; Rosemary Oh; Nagat Bissada; Sazzad M Hossain; Yu-Zhou Yang; Xiao-Jiang Li; Elizabeth M Simpson; Claire-Anne Gutekunst; Blair R Leavitt; Michael R Hayden
Journal: Hum Mol Genet Date: 2003-07-01 Impact factor: 6.150

10. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.

Authors: Jodi L McBride; Ryan L Boudreau; Scott Q Harper; Patrick D Staber; Alex Mas Monteys; Inâs Martins; Brian L Gilmore; Haim Burstein; Richard W Peluso; Barry Polisky; Barrie J Carter; Beverly L Davidson
Journal: Proc Natl Acad Sci U S A Date: 2008-04-08 Impact factor: 11.205

30 in total

Review 1. Recent advances in molecular therapies for neurological disease: triplet repeat disorders.

Authors: Pedro Gonzalez-Alegre
Journal: Hum Mol Genet Date: 2019-10-01 Impact factor: 6.150

Review 2. Cell-based therapies for Huntington's disease.

Authors: Yiju Chen; Richard L Carter; In K Cho; Anthony W S Chan
Journal: Drug Discov Today Date: 2014-03-12 Impact factor: 7.851

Review 3. Therapy development in Huntington disease: From current strategies to emerging opportunities.

Authors: Audrey S Dickey; Albert R La Spada
Journal: Am J Med Genet A Date: 2017-12-08 Impact factor: 2.802

Review 4. Converging pathways in neurodegeneration, from genetics to mechanisms.

Authors: Li Gan; Mark R Cookson; Leonard Petrucelli; Albert R La Spada
Journal: Nat Neurosci Date: 2018-09-26 Impact factor: 24.884

Review 5. Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.

Authors: Megan S Keiser; Holly B Kordasiewicz; Jodi L McBride
Journal: Hum Mol Genet Date: 2015-10-26 Impact factor: 6.150

Review 6. Intrabodies as neuroprotective therapeutics.

Authors: Anne Messer; Shubhada N Joshi
Journal: Neurotherapeutics Date: 2013-07 Impact factor: 7.620

7. Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.

Authors: Chenchen Niu; Thazah P Prakash; Aneeza Kim; John L Quach; Laryssa A Huryn; Yuechen Yang; Edith Lopez; Ali Jazayeri; Gene Hung; Bryce L Sopher; Brian P Brooks; Eric E Swayze; C Frank Bennett; Albert R La Spada
Journal: Sci Transl Med Date: 2018-10-31 Impact factor: 17.956