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Human Gene Therapy
|
July 7, 2022
Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1
Siham Ait Benichou, Dominic Jauvin, Thiéry De Serres-Bérard, et al.
Science Translational Medicine
|
July 29, 2021
Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS
Alyssa N Coyne, Victoria Baskerville, Benjamin L Zaepfel, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 14, 2015
Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy
Laurent P Bogdanik, Melissa A Osborne, Crystal Davis, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
July 3, 2023
The RNA-binding proteins hnRNP H and F regulate splicing of a MYC-dependent HRAS exon in prostate cancer cells
Xinyuan Chen, Harry Taegyun Yang, Beatrice Zhang, et al.
Plos One
|
March 9, 2023
Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn R493X mouse model of frontotemporal dementia
Denise M Smith, Michael L Niehoff, Karen Ling, et al.
Human Molecular Genetics
|
April 21, 2020
ALS-associated genes in SCA2 mouse spinal cord transcriptomes
Daniel R Scoles, Warunee Dansithong, Lance T Pflieger, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
February 2, 2023
Carbogen-Induced Respiratory Acidosis Blocks Experimental Seizures by a Direct and Specific Inhibition of Na<sub>V</sub>1.2 Channels in the Axon Initial Segment of Pyramidal Neurons
Robert J Hatch, Géza Berecki, Nikola Jancovski, et al.
JCI Insight
|
May 17, 2019
An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis
Ilya Gertsman, Joanne Wuu, Melissa McAlonis-Downes, et al.
Acta Neuropathologica
|
November 29, 2023
G<sub>2</sub>C<sub>4</sub> targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
Jeffrey D Rothstein, Victoria Baskerville, Sampath Rapuri, et al.
Journal of Medicinal Chemistry
|
July 18, 2020
Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides
Mehran Nikan, Michael Tanowitz, Chrissa A Dwyer, et al.
Page
of 17
Search research articles
Search
Showing results (51-60 of 163) with videos related to
Sort By:
Page
of 17
Human Gene Therapy
|
July 7, 2022
Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1
Siham Ait Benichou, Dominic Jauvin, Thiéry De Serres-Bérard, et al.
Science Translational Medicine
|
July 29, 2021
Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS
Alyssa N Coyne, Victoria Baskerville, Benjamin L Zaepfel, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
October 14, 2015
Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy
Laurent P Bogdanik, Melissa A Osborne, Crystal Davis, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
July 3, 2023
The RNA-binding proteins hnRNP H and F regulate splicing of a MYC-dependent HRAS exon in prostate cancer cells
Xinyuan Chen, Harry Taegyun Yang, Beatrice Zhang, et al.
Plos One
|
March 9, 2023
Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn R493X mouse model of frontotemporal dementia
Denise M Smith, Michael L Niehoff, Karen Ling, et al.
Human Molecular Genetics
|
April 21, 2020
ALS-associated genes in SCA2 mouse spinal cord transcriptomes
Daniel R Scoles, Warunee Dansithong, Lance T Pflieger, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
February 2, 2023
Carbogen-Induced Respiratory Acidosis Blocks Experimental Seizures by a Direct and Specific Inhibition of Na<sub>V</sub>1.2 Channels in the Axon Initial Segment of Pyramidal Neurons
Robert J Hatch, Géza Berecki, Nikola Jancovski, et al.
JCI Insight
|
May 17, 2019
An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis
Ilya Gertsman, Joanne Wuu, Melissa McAlonis-Downes, et al.
Acta Neuropathologica
|
November 29, 2023
G<sub>2</sub>C<sub>4</sub> targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
Jeffrey D Rothstein, Victoria Baskerville, Sampath Rapuri, et al.
Journal of Medicinal Chemistry
|
July 18, 2020
Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides
Mehran Nikan, Michael Tanowitz, Chrissa A Dwyer, et al.
Page
of 17