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Times Co-cited
Similarity
33
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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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.
Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga, John W Day, Craig Campbell, Anne M Connolly, Susan T Iannaccone, Janbernd Kirschner, Nancy L Kuntz, Kayoko Saito,[...]. N Engl J Med 2018
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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
Richard S Finkel, Eugenio Mercuri, Basil T Darras, Anne M Connolly, Nancy L Kuntz, Janbernd Kirschner, Claudia A Chiriboga, Kayoko Saito, Laurent Servais, Eduardo Tizzano,[...]. N Engl J Med 2017
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Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
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Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
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Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.
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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
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Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.
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LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.
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Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
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Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS.
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Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients.
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Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.
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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
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Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide.
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Biological and clinical changes in premanifest and early stage Huntington's disease in the TRACK-HD study: the 12-month longitudinal analysis.
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8
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.
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Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
Claudia A Chiriboga, Kathryn J Swoboda, Basil T Darras, Susan T Iannaccone, Jacqueline Montes, Darryl C De Vivo, Daniel A Norris, C Frank Bennett, Kathie M Bishop. Neurology 2016
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8
A genetic association study of glutamine-encoding DNA sequence structures, somatic CAG expansion, and DNA repair gene variants, with Huntington disease clinical outcomes.
Marc Ciosi, Alastair Maxwell, Sarah A Cumming, Davina J Hensman Moss, Asma M Alshammari, Michael D Flower, Alexandra Durr, Blair R Leavitt, Raymund A C Roos, Peter Holmans,[...]. EBioMedicine 2019
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14
Evaluation of mutant huntingtin and neurofilament proteins as potential markers in Huntington's disease.
Lauren M Byrne, Filipe B Rodrigues, Eileanor B Johnson, Peter A Wijeratne, Enrico De Vita, Daniel C Alexander, Giuseppe Palermo, Christian Czech, Scott Schobel, Rachael I Scahill,[...]. Sci Transl Med 2018
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8
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.
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8
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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8
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
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The incidence and prevalence of Huntington's disease: a systematic review and meta-analysis.
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In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.
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7
Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.
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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
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Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.
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Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.
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Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.
Alex McCampbell, Tracy Cole, Amy J Wegener, Giulio S Tomassy, Amy Setnicka, Brandon J Farley, Kathleen M Schoch, Mariah L Hoye, Mark Shabsovich, Linhong Sun,[...]. J Clin Invest 2018
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The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington's disease patients.
Andreas Neueder, Christian Landles, Rhia Ghosh, David Howland, Richard H Myers, Richard L M Faull, Sarah J Tabrizi, Gillian P Bates. Sci Rep 2017
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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
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Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
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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.