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Times Cited: 101
Times Cited: 101
Times Cited
Times Co-cited
Similarity
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.
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,[...]. Neuron 2012
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RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.
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. Proc Natl Acad Sci U S A 2005
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Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease.
Jodi L McBride, Mark R Pitzer, Ryan L Boudreau, Brett Dufour, Theodore Hobbs, Sergio R Ojeda, Beverly L Davidson. Mol Ther 2011
Jodi L McBride, Mark R Pitzer, Ryan L Boudreau, Brett Dufour, Theodore Hobbs, Sergio R Ojeda, Beverly L Davidson. Mol Ther 2011
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Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease.
Lisa M Stanek, Sergio P Sardi, Bryan Mastis, Amy R Richards, Christopher M Treleaven, Tatyana Taksir, Kuma Misra, Seng H Cheng, Lamya S Shihabuddin. Hum Gene Ther 2014
Lisa M Stanek, Sergio P Sardi, Bryan Mastis, Amy R Richards, Christopher M Treleaven, Tatyana Taksir, Kuma Misra, Seng H Cheng, Lamya S Shihabuddin. Hum Gene Ther 2014
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Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.
Ryan L Boudreau, Jodi L McBride, Inês Martins, Shihao Shen, Yi Xing, Barrie J Carter, Beverly L Davidson. Mol Ther 2009
Ryan L Boudreau, Jodi L McBride, Inês Martins, Shihao Shen, Yi Xing, Barrie J Carter, Beverly L Davidson. Mol Ther 2009
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A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.
Martin Jinek, Krzysztof Chylinski, Ines Fonfara, Michael Hauer, Jennifer A Doudna, Emmanuelle Charpentier. Science 2012
Martin Jinek, Krzysztof Chylinski, Ines Fonfara, Michael Hauer, Jennifer A Doudna, Emmanuelle Charpentier. Science 2012
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Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.
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. Mol Ther 2011
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CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease.
Su Yang, Renbao Chang, Huiming Yang, Ting Zhao, Yan Hong, Ha Eun Kong, Xiaobo Sun, Zhaohui Qin, Peng Jin, Shihua Li,[...]. J Clin Invest 2017
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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum.
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Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
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Multiplex genome engineering using CRISPR/Cas systems.
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CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo.
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Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.
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Sustained effects of nonallele-specific Huntingtin silencing.
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Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Edith L Pfister, Lori Kennington, Juerg Straubhaar, Sujata Wagh, Wanzhou Liu, Marian DiFiglia, Bernhard Landwehrmeyer, Jean-Paul Vonsattel, Phillip D Zamore, Neil Aronin. Curr Biol 2009
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Huntington disease: natural history, biomarkers and prospects for therapeutics.
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Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease.
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Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease.
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Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes.
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In vivo genome editing using Staphylococcus aureus Cas9.
F Ann Ran, Le Cong, Winston X Yan, David A Scott, Jonathan S Gootenberg, Andrea J Kriz, Bernd Zetsche, Ophir Shalem, Xuebing Wu, Kira S Makarova,[...]. Nature 2015
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Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice.
Edgardo Rodriguez-Lebron, Eileen M Denovan-Wright, Kevin Nash, Alfred S Lewin, Ronald J Mandel. Mol Ther 2005
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Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease.
Kirupa Sathasivam, Andreas Neueder, Theresa A Gipson, Christian Landles, Agnesska C Benjamin, Marie K Bondulich, Donna L Smith, Richard L M Faull, Raymund A C Roos, David Howland,[...]. Proc Natl Acad Sci U S A 2013
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CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes.
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Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers.
Isaac B Hilton, Anthony M D'Ippolito, Christopher M Vockley, Pratiksha I Thakore, Gregory E Crawford, Timothy E Reddy, Charles A Gersbach. Nat Biotechnol 2015
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Therapies targeting DNA and RNA in Huntington's disease.
Edward J Wild, Sarah J Tabrizi. Lancet Neurol 2017
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Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells.
David Mittelman, Christopher Moye, Jason Morton, Kristen Sykoudis, Yunfu Lin, Dana Carroll, John H Wilson. Proc Natl Acad Sci U S A 2009
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Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.
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Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.
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Breaking the code of DNA binding specificity of TAL-type III effectors.
Jens Boch, Heidi Scholze, Sebastian Schornack, Angelika Landgraf, Simone Hahn, Sabine Kay, Thomas Lahaye, Anja Nickstadt, Ulla Bonas. Science 2009
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Optical control of mammalian endogenous transcription and epigenetic states.
Silvana Konermann, Mark D Brigham, Alexandro Trevino, Patrick D Hsu, Matthias Heidenreich, Le Cong, Randall J Platt, David A Scott, George M Church, Feng Zhang. Nature 2013
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RNA-guided human genome engineering via Cas9.
Prashant Mali, Luhan Yang, Kevin M Esvelt, John Aach, Marc Guell, James E DiCarlo, Julie E Norville, George M Church. Science 2013
Prashant Mali, Luhan Yang, Kevin M Esvelt, John Aach, Marc Guell, James E DiCarlo, Julie E Norville, George M Church. Science 2013
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AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease.
Nicholas R Franich, Helen L Fitzsimons, Dahna M Fong, Matthias Klugmann, Matthew J During, Deborah Young. Mol Ther 2008
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In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.
Amber L Southwell, Niels H Skotte, Holly B Kordasiewicz, Michael E Østergaard, Andrew T Watt, Jeffrey B Carroll, Crystal N Doty, Erika B Villanueva, Eugenia Petoukhov, Kuljeet Vaid,[...]. Mol Ther 2014
Amber L Southwell, Niels H Skotte, Holly B Kordasiewicz, Michael E Østergaard, Andrew T Watt, Jeffrey B Carroll, Crystal N Doty, Erika B Villanueva, Eugenia Petoukhov, Kuljeet Vaid,[...]. Mol Ther 2014
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Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice.
Carmen Agustín-Pavón, Michal Mielcarek, Mireia Garriga-Canut, Mark Isalan. Mol Neurodegener 2016
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Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
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Molecular mechanisms and potential therapeutical targets in Huntington's disease.
Chiara Zuccato, Marta Valenza, Elena Cattaneo. Physiol Rev 2010
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A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.
Maria Stella Lombardi, Leonie Jaspers, Christine Spronkmans, Cinzia Gellera, Franco Taroni, Emilio Di Maria, Stefano Di Donato, William F Kaemmerer. Exp Neurol 2009
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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.
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Therapeutic genome editing: prospects and challenges.
David Benjamin Turitz Cox, Randall Jeffrey Platt, Feng Zhang. Nat Med 2015
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Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients.
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14
Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts.
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Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis.
Guohao Wang, Xudong Liu, Marta A Gaertig, Shihua Li, Xiao-Jiang Li. Proc Natl Acad Sci U S A 2016
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Huntington disease.
Gillian P Bates, Ray Dorsey, James F Gusella, Michael R Hayden, Chris Kay, Blair R Leavitt, Martha Nance, Christopher A Ross, Rachael I Scahill, Ronald Wetzel,[...]. Nat Rev Dis Primers 2015
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Targeting Huntingtin Expression in Patients with Huntington's Disease.
Sarah J Tabrizi, Blair R Leavitt, G Bernhard Landwehrmeyer, Edward J Wild, Carsten Saft, Roger A Barker, Nick F Blair, David Craufurd, Josef Priller, Hugh Rickards,[...]. N Engl J Med 2019
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Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.
Brett D Dufour, Catherine A Smith, Randall L Clark, Timothy R Walker, Jodi L McBride. Mol Ther 2014
Brett D Dufour, Catherine A Smith, Randall L Clark, Timothy R Walker, Jodi L McBride. Mol Ther 2014
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A TALE nuclease architecture for efficient genome editing.
Jeffrey C Miller, Siyuan Tan, Guijuan Qiao, Kyle A Barlow, Jianbin Wang, Danny F Xia, Xiangdong Meng, David E Paschon, Elo Leung, Sarah J Hinkley,[...]. Nat Biotechnol 2011
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13
Co-cited is the co-citation frequency, indicating how many articles cite the article together with the query article. Similarity is the co-citation as percentage of the times cited of the query article or the article in the search results, whichever is the lowest. These numbers are calculated for the last 100 citations when articles are cited more than 100 times.