271 related articles for article (PubMed ID: 18549309)
1. Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts.
van Bilsen PH; Jaspers L; Lombardi MS; Odekerken JC; Burright EN; Kaemmerer WF
Hum Gene Ther; 2008 Jul; 19(7):710-9. PubMed ID: 18549309
[TBL] [Abstract][Full Text] [Related]
2. Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain.
Liu W; Chaurette J; Pfister EL; Kennington LA; Chase KO; Bullock J; Vonsattel JP; Faull RL; Macdonald D; DiFiglia M; Zamore PD; Aronin N
J Huntingtons Dis; 2013; 2(4):491-500. PubMed ID: 25062733
[TBL] [Abstract][Full Text] [Related]
3. Sustained effects of nonallele-specific Huntingtin silencing.
Drouet V; Perrin V; Hassig R; Dufour N; Auregan G; Alves S; Bonvento G; Brouillet E; Luthi-Carter R; Hantraye P; Déglon N
Ann Neurol; 2009 Mar; 65(3):276-85. PubMed ID: 19334076
[TBL] [Abstract][Full Text] [Related]
4. Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells.
Drouet V; Ruiz M; Zala D; Feyeux M; Auregan G; Cambon K; Troquier L; Carpentier J; Aubert S; Merienne N; Bourgois-Rocha F; Hassig R; Rey M; Dufour N; Saudou F; Perrier AL; Hantraye P; Déglon N
PLoS One; 2014; 9(6):e99341. PubMed ID: 24926995
[TBL] [Abstract][Full Text] [Related]
5. Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Pfister EL; Kennington L; Straubhaar J; Wagh S; Liu W; DiFiglia M; Landwehrmeyer B; Vonsattel JP; Zamore PD; Aronin N
Curr Biol; 2009 May; 19(9):774-8. PubMed ID: 19361997
[TBL] [Abstract][Full Text] [Related]
6. 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. PubMed ID: 21971427
[TBL] [Abstract][Full Text] [Related]
7. A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.
Lombardi MS; Jaspers L; Spronkmans C; Gellera C; Taroni F; Di Maria E; Donato SD; Kaemmerer WF
Exp Neurol; 2009 Jun; 217(2):312-9. PubMed ID: 19289118
[TBL] [Abstract][Full Text] [Related]
8. Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs.
Hu J; Liu J; Yu D; Aiba Y; Lee S; Pendergraff H; Boubaker J; Artates JW; Lagier-Tourenne C; Lima WF; Swayze EE; Prakash TP; Corey DR
Nucleic Acid Ther; 2014 Jun; 24(3):199-209. PubMed ID: 24694346
[TBL] [Abstract][Full Text] [Related]
9. Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.
Hu J; Liu J; Corey DR
Chem Biol; 2010 Nov; 17(11):1183-8. PubMed ID: 21095568
[TBL] [Abstract][Full Text] [Related]
10. Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Miller JRC; Pfister EL; Liu W; Andre R; Träger U; Kennington LA; Lo K; Dijkstra S; Macdonald D; Ostroff G; Aronin N; Tabrizi SJ
Sci Rep; 2017 Apr; 7():46740. PubMed ID: 28436437
[TBL] [Abstract][Full Text] [Related]
11. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease.
McBride JL; Pitzer MR; Boudreau RL; Dufour B; Hobbs T; Ojeda SR; Davidson BL
Mol Ther; 2011 Dec; 19(12):2152-62. PubMed ID: 22031240
[TBL] [Abstract][Full Text] [Related]
12. Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.
Shin JW; Kim KH; Chao MJ; Atwal RS; Gillis T; MacDonald ME; Gusella JF; Lee JM
Hum Mol Genet; 2016 Oct; 25(20):4566-4576. PubMed ID: 28172889
[TBL] [Abstract][Full Text] [Related]
13. Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.
Boudreau RL; McBride JL; Martins I; Shen S; Xing Y; Carter BJ; Davidson BL
Mol Ther; 2009 Jun; 17(6):1053-63. PubMed ID: 19240687
[TBL] [Abstract][Full Text] [Related]
14. Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum.
Grondin R; Kaytor MD; Ai Y; Nelson PT; Thakker DR; Heisel J; Weatherspoon MR; Blum JL; Burright EN; Zhang Z; Kaemmerer WF
Brain; 2012 Apr; 135(Pt 4):1197-209. PubMed ID: 22252996
[TBL] [Abstract][Full Text] [Related]
15. AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Miniarikova J; Zimmer V; Martier R; Brouwers CC; Pythoud C; Richetin K; Rey M; Lubelski J; Evers MM; van Deventer SJ; Petry H; Déglon N; Konstantinova P
Gene Ther; 2017 Oct; 24(10):630-639. PubMed ID: 28771234
[TBL] [Abstract][Full Text] [Related]
16. Using non-coding small RNAs to develop therapies for Huntington's disease.
Zhang Y; Friedlander RM
Gene Ther; 2011 Dec; 18(12):1139-49. PubMed ID: 22158031
[TBL] [Abstract][Full Text] [Related]
17. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo.
Huang B; Schiefer J; Sass C; Landwehrmeyer GB; Kosinski CM; Kochanek S
Hum Gene Ther; 2007 Apr; 18(4):303-11. PubMed ID: 17472569
[TBL] [Abstract][Full Text] [Related]
18. Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.
Kay C; Collins JA; Skotte NH; Southwell AL; Warby SC; Caron NS; Doty CN; Nguyen B; Griguoli A; Ross CJ; Squitieri F; Hayden MR
Mol Ther; 2015 Nov; 23(11):1759-1771. PubMed ID: 26201449
[TBL] [Abstract][Full Text] [Related]
19. Widespread suppression of huntingtin with convection-enhanced delivery of siRNA.
Stiles DK; Zhang Z; Ge P; Nelson B; Grondin R; Ai Y; Hardy P; Nelson PT; Guzaev AP; Butt MT; Charisse K; Kosovrasti V; Tchangov L; Meys M; Maier M; Nechev L; Manoharan M; Kaemmerer WF; Gwost D; Stewart GR; Gash DM; Sah DW
Exp Neurol; 2012 Jan; 233(1):463-71. PubMed ID: 22119622
[TBL] [Abstract][Full Text] [Related]
20. Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease.
Stanek LM; Sardi SP; Mastis B; Richards AR; Treleaven CM; Taksir T; Misra K; Cheng SH; Shihabuddin LS
Hum Gene Ther; 2014 May; 25(5):461-74. PubMed ID: 24484067
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]