Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.
Jun Wan Shin, Kyung-Hee Kim, Michael J Chao, Ranjit S Atwal, Tammy Gillis, Marcy E MacDonald, James F Gusella, Jong-Min Lee. Hum Mol Genet 2016
Times Cited: 130
Times Cited: 130
Times Cited
Times Co-cited
Similarity
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo.
Alex Mas Monteys, Shauna A Ebanks, Megan S Keiser, Beverly L Davidson. Mol Ther 2017
Alex Mas Monteys, Shauna A Ebanks, Megan S Keiser, Beverly L Davidson. Mol Ther 2017
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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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Multiplex genome engineering using CRISPR/Cas systems.
Le Cong, F Ann Ran, David Cox, Shuailiang Lin, Robert Barretto, Naomi Habib, Patrick D Hsu, Xuebing Wu, Wenyan Jiang, Luciano A Marraffini,[...]. Science 2013
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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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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
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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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Therapies targeting DNA and RNA in Huntington's disease.
Edward J Wild, Sarah J Tabrizi. Lancet Neurol 2017
Edward J Wild, Sarah J Tabrizi. Lancet Neurol 2017
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Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells.
Xiaohong Xu, Yilin Tay, Bernice Sim, Su-In Yoon, Yihui Huang, Jolene Ooi, Kagistia Hana Utami, Amin Ziaei, Bryan Ng, Carola Radulescu,[...]. Stem Cell Reports 2017
Xiaohong Xu, Yilin Tay, Bernice Sim, Su-In Yoon, Yihui Huang, Jolene Ooi, Kagistia Hana Utami, Amin Ziaei, Bryan Ng, Carola Radulescu,[...]. Stem Cell Reports 2017
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Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases.
Magdalena Dabrowska, Wojciech Juzwa, Wlodzimierz J Krzyzosiak, Marta Olejniczak. Front Neurosci 2018
Magdalena Dabrowska, Wojciech Juzwa, Wlodzimierz J Krzyzosiak, Marta Olejniczak. Front Neurosci 2018
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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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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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CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington's Disease.
Nivya Kolli, Ming Lu, Panchanan Maiti, Julien Rossignol, Gray L Dunbar. Int J Mol Sci 2017
Nivya Kolli, Ming Lu, Panchanan Maiti, Julien Rossignol, Gray L Dunbar. Int J Mol Sci 2017
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Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells.
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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
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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
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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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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Development and applications of CRISPR-Cas9 for genome engineering.
Patrick D Hsu, Eric S Lander, Feng Zhang. Cell 2014
Patrick D Hsu, Eric S Lander, Feng Zhang. Cell 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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Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.
Alexis C Komor, Yongjoo B Kim, Michael S Packer, John A Zuris, David R Liu. Nature 2016
Alexis C Komor, Yongjoo B Kim, Michael S Packer, John A Zuris, David R Liu. Nature 2016
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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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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
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Genome engineering using the CRISPR-Cas9 system.
F Ann Ran, Patrick D Hsu, Jason Wright, Vineeta Agarwala, David A Scott, Feng Zhang. Nat Protoc 2013
F Ann Ran, Patrick D Hsu, Jason Wright, Vineeta Agarwala, David A Scott, Feng Zhang. Nat Protoc 2013
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DNA targeting specificity of RNA-guided Cas9 nucleases.
Patrick D Hsu, David A Scott, Joshua A Weinstein, F Ann Ran, Silvana Konermann, Vineeta Agarwala, Yinqing Li, Eli J Fine, Xuebing Wu, Ophir Shalem,[...]. Nat Biotechnol 2013
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High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.
Benjamin P Kleinstiver, Vikram Pattanayak, Michelle S Prew, Shengdar Q Tsai, Nhu T Nguyen, Zongli Zheng, J Keith Joung. Nature 2016
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Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice.
Mireia Garriga-Canut, Carmen Agustín-Pavón, Frank Herrmann, Aurora Sánchez, Mara Dierssen, Cristina Fillat, Mark Isalan. Proc Natl Acad Sci U S A 2012
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Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system.
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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
Guohao Wang, Xudong Liu, Marta A Gaertig, Shihua Li, Xiao-Jiang Li. Proc Natl Acad Sci U S A 2016
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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum.
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Therapeutic genome editing: prospects and challenges.
David Benjamin Turitz Cox, Randall Jeffrey Platt, Feng Zhang. Nat Med 2015
David Benjamin Turitz Cox, Randall Jeffrey Platt, Feng Zhang. Nat Med 2015
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In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.
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Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue.
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Rationally engineered Cas9 nucleases with improved specificity.
Ian M Slaymaker, Linyi Gao, Bernd Zetsche, David A Scott, Winston X Yan, Feng Zhang. Science 2016
Ian M Slaymaker, Linyi Gao, Bernd Zetsche, David A Scott, Winston X Yan, Feng Zhang. Science 2016
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CRISPR/Cas9-Targeted Deletion of Polyglutamine in Spinocerebellar Ataxia Type 3-Derived Induced Pluripotent Stem Cells.
Shuming Ouyang, Yingjun Xie, Zeyu Xiong, Yi Yang, Yexing Xian, Zhanhui Ou, Bing Song, Yuchang Chen, Yuhuan Xie, Haoxian Li,[...]. Stem Cells Dev 2018
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Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.
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Sustained effects of nonallele-specific Huntingtin silencing.
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Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.
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CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model.
Freja K Ekman, David S Ojala, Maroof M Adil, Paola A Lopez, David V Schaffer, Thomas Gaj. Mol Ther Nucleic Acids 2019
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Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice.
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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.
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Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
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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.
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Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.
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Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements.
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Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.
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AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
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CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.
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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.