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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 8, 2014
In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides
Amber L Southwell, Niels H Skotte, Holly B Kordasiewicz, et al.
Plos One
|
September 11, 2014
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients
Niels H Skotte, Amber L Southwell, Michael E Østergaard, et al.
Biorxiv : the Preprint Server for Biology
|
June 19, 2024
Identification of genetic modifiers of Huntington's disease somatic CAG repeat instability by in vivo CRISPR-Cas9 genome editing
Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, et al.
Molecular Therapy. Nucleic Acids
|
February 2, 2026
Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model
Velvet L Smith, Bereket Z Gidi, Robert M Bragg, et al.
Journal of Parkinson'S Disease
|
February 20, 2025
Improving recruitment and retention of people with Parkinson's disease to clinical studies: A scoping review
Rebecca Petty, Veena Agarwal, Jennifer Allison, et al.
Nature Genetics
|
January 22, 2025
In vivo CRISPR-Cas9 genome editing in mice identifies genetic modifiers of somatic CAG repeat instability in Huntington's disease
Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, et al.
Communications Biology
|
December 9, 2021
Huntingtin structure is orchestrated by HAP40 and shows a polyglutamine expansion-specific interaction with exon 1
Rachel J Harding, Justin C Deme, Johannes F Hevler, et al.
Neurobiology of Aging
|
September 19, 2016
Rare variants analysis of cutaneous malignant melanoma genes in Parkinson's disease
S J Lubbe, V Escott-Price, A Brice, et al.
BMJ Neurology Open
|
July 6, 2026
Identifying and predicting fast versus slow Parkinson's disease motor progressors using clinical and digital data
Timothee Aubourg, Katarina M Gunter, Christine Lo, et al.
Movement Disorders : Official Journal of the Movement Disorder Society
|
December 23, 2023
Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials
Cristina Gonzalez-Robles, Michèle Bartlett, Matthew Burnell, et al.
Page
of 58
Search research articles
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Showing results (551-560 of 574) with videos related to
Sort By:
Page
of 58
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 8, 2014
In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides
Amber L Southwell, Niels H Skotte, Holly B Kordasiewicz, et al.
Plos One
|
September 11, 2014
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients
Niels H Skotte, Amber L Southwell, Michael E Østergaard, et al.
Biorxiv : the Preprint Server for Biology
|
June 19, 2024
Identification of genetic modifiers of Huntington's disease somatic CAG repeat instability by in vivo CRISPR-Cas9 genome editing
Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, et al.
Molecular Therapy. Nucleic Acids
|
February 2, 2026
Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model
Velvet L Smith, Bereket Z Gidi, Robert M Bragg, et al.
Journal of Parkinson'S Disease
|
February 20, 2025
Improving recruitment and retention of people with Parkinson's disease to clinical studies: A scoping review
Rebecca Petty, Veena Agarwal, Jennifer Allison, et al.
Nature Genetics
|
January 22, 2025
In vivo CRISPR-Cas9 genome editing in mice identifies genetic modifiers of somatic CAG repeat instability in Huntington's disease
Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, et al.
Communications Biology
|
December 9, 2021
Huntingtin structure is orchestrated by HAP40 and shows a polyglutamine expansion-specific interaction with exon 1
Rachel J Harding, Justin C Deme, Johannes F Hevler, et al.
Neurobiology of Aging
|
September 19, 2016
Rare variants analysis of cutaneous malignant melanoma genes in Parkinson's disease
S J Lubbe, V Escott-Price, A Brice, et al.
BMJ Neurology Open
|
July 6, 2026
Identifying and predicting fast versus slow Parkinson's disease motor progressors using clinical and digital data
Timothee Aubourg, Katarina M Gunter, Christine Lo, et al.
Movement Disorders : Official Journal of the Movement Disorder Society
|
December 23, 2023
Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials
Cristina Gonzalez-Robles, Michèle Bartlett, Matthew Burnell, et al.
Page
of 58