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, Anne Rosser, Holly B Kordasiewicz, Christian Czech, Eric E Swayze, Daniel A Norris, Tiffany Baumann, Irene Gerlach, Scott A Schobel, Erika Paz, Anne V Smith, C Frank Bennett, Roger M Lane. N Engl J Med 2019
Times Cited: 287
Times Cited: 287
Times Cited
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
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
22
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
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
17
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
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
17
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Timothy Miller, Merit Cudkowicz, Pamela J Shaw, Peter M Andersen, Nazem Atassi, Robert C Bucelli, Angela Genge, Jonathan Glass, Shafeeq Ladha, Albert L Ludolph,[...]. N Engl J Med 2020
Timothy Miller, Merit Cudkowicz, Pamela J Shaw, Peter M Andersen, Nazem Atassi, Robert C Bucelli, Angela Genge, Jonathan Glass, Shafeeq Ladha, Albert L Ludolph,[...]. N Engl J Med 2020
17
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
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
16
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
Timothy M Miller, Alan Pestronk, William David, Jeffrey Rothstein, Ericka Simpson, Stanley H Appel, Patricia L Andres, Katy Mahoney, Peggy Allred, Katie Alexander,[...]. Lancet Neurol 2013
Timothy M Miller, Alan Pestronk, William David, Jeffrey Rothstein, Ericka Simpson, Stanley H Appel, Patricia L Andres, Katy Mahoney, Peggy Allred, Katie Alexander,[...]. Lancet Neurol 2013
15
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
Richard S Finkel, Claudia A Chiriboga, Jiri Vajsar, John W Day, Jacqueline Montes, Darryl C De Vivo, Mason Yamashita, Frank Rigo, Gene Hung, Eugene Schneider,[...]. Lancet 2016
Richard S Finkel, Claudia A Chiriboga, Jiri Vajsar, John W Day, Jacqueline Montes, Darryl C De Vivo, Mason Yamashita, Frank Rigo, Gene Hung, Eugene Schneider,[...]. Lancet 2016
13
CAG Repeat Not Polyglutamine Length Determines Timing of Huntington's Disease Onset.
. Cell 2019
. Cell 2019
12
Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.
C Frank Bennett, Adrian R Krainer, Don W Cleveland. Annu Rev Neurosci 2019
C Frank Bennett, Adrian R Krainer, Don W Cleveland. Annu Rev Neurosci 2019
12
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
Bryan Zeitler, Steven Froelich, Kimberly Marlen, David A Shivak, Qi Yu, Davis Li, Jocelynn R Pearl, Jeffrey C Miller, Lei Zhang, David E Paschon,[...]. Nat Med 2019
Bryan Zeitler, Steven Froelich, Kimberly Marlen, David A Shivak, Qi Yu, Davis Li, Jocelynn R Pearl, Jeffrey C Miller, Lei Zhang, David E Paschon,[...]. Nat Med 2019
16
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
11
Huntingtin Lowering Strategies for Disease Modification in Huntington's Disease.
Sarah J Tabrizi, Rhia Ghosh, Blair R Leavitt. Neuron 2019
Sarah J Tabrizi, Rhia Ghosh, Blair R Leavitt. Neuron 2019
13
Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease.
Amber L Southwell, Holly B Kordasiewicz, Douglas Langbehn, Niels H Skotte, Matthew P Parsons, Erika B Villanueva, Nicholas S Caron, Michael E Østergaard, Lisa M Anderson, Yuanyun Xie,[...]. Sci Transl Med 2018
Amber L Southwell, Holly B Kordasiewicz, Douglas Langbehn, Niels H Skotte, Matthew P Parsons, Erika B Villanueva, Nicholas S Caron, Michael E Østergaard, Lisa M Anderson, Yuanyun Xie,[...]. Sci Transl Med 2018
16
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Julia F Alterman, Bruno M D C Godinho, Matthew R Hassler, Chantal M Ferguson, Dimas Echeverria, Ellen Sapp, Reka A Haraszti, Andrew H Coles, Faith Conroy, Rachael Miller,[...]. Nat Biotechnol 2019
Julia F Alterman, Bruno M D C Godinho, Matthew R Hassler, Chantal M Ferguson, Dimas Echeverria, Ellen Sapp, Reka A Haraszti, Andrew H Coles, Faith Conroy, Rachael Miller,[...]. Nat Biotechnol 2019
19
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Jerry R Mendell, Samiah Al-Zaidy, Richard Shell, W Dave Arnold, Louise R Rodino-Klapac, Thomas W Prior, Linda Lowes, Lindsay Alfano, Katherine Berry, Kathleen Church,[...]. N Engl J Med 2017
Jerry R Mendell, Samiah Al-Zaidy, Richard Shell, W Dave Arnold, Louise R Rodino-Klapac, Thomas W Prior, Linda Lowes, Lindsay Alfano, Katherine Berry, Kathleen Church,[...]. N Engl J Med 2017
10
Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.
Daniel R Scoles, Pratap Meera, Matthew D Schneider, Sharan Paul, Warunee Dansithong, Karla P Figueroa, Gene Hung, Frank Rigo, C Frank Bennett, Thomas S Otis,[...]. Nature 2017
Daniel R Scoles, Pratap Meera, Matthew D Schneider, Sharan Paul, Warunee Dansithong, Karla P Figueroa, Gene Hung, Frank Rigo, C Frank Bennett, Thomas S Otis,[...]. Nature 2017
9
Neuropathological classification of Huntington's disease.
J P Vonsattel, R H Myers, T J Stevens, R J Ferrante, E D Bird, E P Richardson. J Neuropathol Exp Neurol 1985
J P Vonsattel, R H Myers, T J Stevens, R J Ferrante, E D Bird, E P Richardson. J Neuropathol Exp Neurol 1985
9
LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.
Hien Tran Zhao, Neena John, Vedad Delic, Karli Ikeda-Lee, Aneeza Kim, Andreas Weihofen, Eric E Swayze, Holly B Kordasiewicz, Andrew B West, Laura A Volpicelli-Daley. Mol Ther Nucleic Acids 2017
Hien Tran Zhao, Neena John, Vedad Delic, Karli Ikeda-Lee, Aneeza Kim, Andreas Weihofen, Eric E Swayze, Holly B Kordasiewicz, Andrew B West, Laura A Volpicelli-Daley. Mol Ther Nucleic Acids 2017
10
Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
Nicholas S Caron, Amber L Southwell, Cynthia C Brouwers, Louisa Dal Cengio, Yuanyun Xie, Hailey Findlay Black, Lisa M Anderson, Seunghyun Ko, Xiang Zhu, Sander J van Deventer,[...]. Nucleic Acids Res 2020
Nicholas S Caron, Amber L Southwell, Cynthia C Brouwers, Louisa Dal Cengio, Yuanyun Xie, Hailey Findlay Black, Lisa M Anderson, Seunghyun Ko, Xiang Zhu, Sander J van Deventer,[...]. Nucleic Acids Res 2020
37
Unified Huntington's Disease Rating Scale: reliability and consistency. Huntington Study Group.
. Mov Disord 1996
. Mov Disord 1996
9
Huntington disease: natural history, biomarkers and prospects for therapeutics.
Christopher A Ross, Elizabeth H Aylward, Edward J Wild, Douglas R Langbehn, Jeffrey D Long, John H Warner, Rachael I Scahill, Blair R Leavitt, Julie C Stout, Jane S Paulsen,[...]. Nat Rev Neurol 2014
Christopher A Ross, Elizabeth H Aylward, Edward J Wild, Douglas R Langbehn, Jeffrey D Long, John H Warner, Rachael I Scahill, Blair R Leavitt, Julie C Stout, Jane S Paulsen,[...]. Nat Rev Neurol 2014
9
Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS.
Mariely DeJesus-Hernandez, Ian R Mackenzie, Bradley F Boeve, Adam L Boxer, Matt Baker, Nicola J Rutherford, Alexandra M Nicholson, NiCole A Finch, Heather Flynn, Jennifer Adamson,[...]. Neuron 2011
Mariely DeJesus-Hernandez, Ian R Mackenzie, Bradley F Boeve, Adam L Boxer, Matt Baker, Nicola J Rutherford, Alexandra M Nicholson, NiCole A Finch, Heather Flynn, Jennifer Adamson,[...]. Neuron 2011
9
Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients.
Edward J Wild, Roberto Boggio, Douglas Langbehn, Nicola Robertson, Salman Haider, James R C Miller, Henrik Zetterberg, Blair R Leavitt, Rainer Kuhn, Sarah J Tabrizi,[...]. J Clin Invest 2015
Edward J Wild, Roberto Boggio, Douglas Langbehn, Nicola Robertson, Salman Haider, James R C Miller, Henrik Zetterberg, Blair R Leavitt, Rainer Kuhn, Sarah J Tabrizi,[...]. J Clin Invest 2015
9
Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7.
Chenchen Niu, Thazah P Prakash, Aneeza Kim, John L Quach, Laryssa A Huryn, Yuechen Yang, Edith Lopez, Ali Jazayeri, Gene Hung, Bryce L Sopher,[...]. Sci Transl Med 2018
Chenchen Niu, Thazah P Prakash, Aneeza Kim, John L Quach, Laryssa A Huryn, Yuechen Yang, Edith Lopez, Ali Jazayeri, Gene Hung, Bryce L Sopher,[...]. Sci Transl Med 2018
19
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
Jinkuk Kim, Chunguang Hu, Christelle Moufawad El Achkar, Lauren E Black, Julie Douville, Austin Larson, Mary K Pendergast, Sara F Goldkind, Eunjung A Lee, Ashley Kuniholm,[...]. N Engl J Med 2019
Jinkuk Kim, Chunguang Hu, Christelle Moufawad El Achkar, Lauren E Black, Julie Douville, Austin Larson, Mary K Pendergast, Sara F Goldkind, Eunjung A Lee, Ashley Kuniholm,[...]. N Engl J Med 2019
8
Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide.
P C Zamecnik, M L Stephenson. Proc Natl Acad Sci U S A 1978
P C Zamecnik, M L Stephenson. Proc Natl Acad Sci U S A 1978
8
Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.
Sarah L DeVos, Rebecca L Miller, Kathleen M Schoch, Brandon B Holmes, Carey S Kebodeaux, Amy J Wegener, Guo Chen, Tao Shen, Hien Tran, Brandon Nichols,[...]. Sci Transl Med 2017
Sarah L DeVos, Rebecca L Miller, Kathleen M Schoch, Brandon B Holmes, Carey S Kebodeaux, Amy J Wegener, Guo Chen, Tao Shen, Hien Tran, Brandon Nichols,[...]. Sci Transl Med 2017
8
RNA-Targeted Therapeutics.
Stanley T Crooke, Joseph L Witztum, C Frank Bennett, Brenda F Baker. Cell Metab 2018
Stanley T Crooke, Joseph L Witztum, C Frank Bennett, Brenda F Baker. Cell Metab 2018
8
Biological and clinical changes in premanifest and early stage Huntington's disease in the TRACK-HD study: the 12-month longitudinal analysis.
Sarah J Tabrizi, Rachael I Scahill, Alexandra Durr, Raymund Ac Roos, Blair R Leavitt, Rebecca Jones, G Bernhard Landwehrmeyer, Nick C Fox, Hans Johnson, Stephen L Hicks,[...]. Lancet Neurol 2011
Sarah J Tabrizi, Rachael I Scahill, Alexandra Durr, Raymund Ac Roos, Blair R Leavitt, Rebecca Jones, G Bernhard Landwehrmeyer, Nick C Fox, Hans Johnson, Stephen L Hicks,[...]. Lancet Neurol 2011
8
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
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
8
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
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
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
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
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
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
13
Identification of Genetic Factors that Modify Clinical Onset of Huntington's Disease.
. Cell 2015
. Cell 2015
8
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
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
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
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
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
Jodi L McBride, Mark R Pitzer, Ryan L Boudreau, Brett Dufour, Theodore Hobbs, Sergio R Ojeda, Beverly L Davidson. Mol Ther 2011
8
The incidence and prevalence of Huntington's disease: a systematic review and meta-analysis.
Tamara Pringsheim, Katie Wiltshire, Lundy Day, Jonathan Dykeman, Thomas Steeves, Nathalie Jette. Mov Disord 2012
Tamara Pringsheim, Katie Wiltshire, Lundy Day, Jonathan Dykeman, Thomas Steeves, Nathalie Jette. Mov Disord 2012
7
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
7
Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.
Jillian Friedrich, Holly B Kordasiewicz, Brennon O'Callaghan, Hillary P Handler, Carmen Wagener, Lisa Duvick, Eric E Swayze, Orion Rainwater, Bente Hofstra, Michael Benneyworth,[...]. JCI Insight 2018
Jillian Friedrich, Holly B Kordasiewicz, Brennon O'Callaghan, Hillary P Handler, Carmen Wagener, Lisa Duvick, Eric E Swayze, Orion Rainwater, Bente Hofstra, Michael Benneyworth,[...]. JCI Insight 2018
14
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.
Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J Wells,[...]. Lancet 2011
Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J Wells,[...]. Lancet 2011
7
Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.
Hayley S McLoughlin, Lauren R Moore, Ravi Chopra, Robert Komlo, Megan McKenzie, Kate G Blumenstein, Hien Zhao, Holly B Kordasiewicz, Vikram G Shakkottai, Henry L Paulson. Ann Neurol 2018
Hayley S McLoughlin, Lauren R Moore, Ravi Chopra, Robert Komlo, Megan McKenzie, Kate G Blumenstein, Hien Zhao, Holly B Kordasiewicz, Vikram G Shakkottai, Henry L Paulson. Ann Neurol 2018
9
Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.
Jiaxin Hu, Masayuki Matsui, Keith T Gagnon, Jacob C Schwartz, Sylvie Gabillet, Khalil Arar, Jun Wu, Ilya Bezprozvanny, David R Corey. Nat Biotechnol 2009
Jiaxin Hu, Masayuki Matsui, Keith T Gagnon, Jacob C Schwartz, Sylvie Gabillet, Khalil Arar, Jun Wu, Ilya Bezprozvanny, David R Corey. Nat Biotechnol 2009
7
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
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
8
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
Andreas Neueder, Christian Landles, Rhia Ghosh, David Howland, Richard H Myers, Richard L M Faull, Sarah J Tabrizi, Gillian P Bates. Sci Rep 2017
9
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
7
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
Dongbo Yu, Hannah Pendergraff, Jing Liu, Holly B Kordasiewicz, Don W Cleveland, Eric E Swayze, Walt F Lima, Stanley T Crooke, Thazha P Prakash, David R Corey. Cell 2012
Dongbo Yu, Hannah Pendergraff, Jing Liu, Holly B Kordasiewicz, Don W Cleveland, Eric E Swayze, Walt F Lima, Stanley T Crooke, Thazha P Prakash, David R Corey. Cell 2012
7
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides.
Richard S Geary, Daniel Norris, Rosie Yu, C Frank Bennett. Adv Drug Deliv Rev 2015
Richard S Geary, Daniel Norris, Rosie Yu, C Frank Bennett. Adv Drug Deliv Rev 2015
7
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.