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Molecular Therapy. Nucleic Acids
|
February 12, 2021
Targeting the 5' untranslated region of <i>SMN2</i> as a therapeutic strategy for spinal muscular atrophy
Audrey M Winkelsas, Christopher Grunseich, George G Harmison, et al.
EMBO Molecular Medicine
|
September 19, 2023
PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice
Anna J Kordala, Jessica Stoodley, Nina Ahlskog, et al.
Journal of Neurology
|
June 28, 2011
A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results
Francesco Saccà, Mario Quarantelli, Carlo Rinaldi, et al.
Nucleic Acids Research
|
January 23, 2025
Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs
Antonio Garcia-Guerra, Chaitra Sathyaprakash, Olivier G de Jong, et al.
Human Molecular Genetics
|
March 11, 2016
A small-molecule Nrf1 and Nrf2 activator mitigates polyglutamine toxicity in spinal and bulbar muscular atrophy
Laura C Bott, Nisha M Badders, Ke-Lian Chen, et al.
American Journal of Human Genetics
|
December 11, 2012
Cowchock syndrome is associated with a mutation in apoptosis-inducing factor
Carlo Rinaldi, Christopher Grunseich, Irina F Sevrioukova, et al.
Journal of Neurology
|
April 14, 2009
Two novel CYP7B1 mutations in Italian families with SPG5: a clinical and genetic study
Chiara Criscuolo, Alessandro Filla, Giovanni Coppola, et al.
Cells
|
February 6, 2020
Polyglutamine-Expanded Androgen Receptor Alteration of Skeletal Muscle Homeostasis and Myonuclear Aggregation Are Affected by Sex, Age and Muscle Metabolism
Mathilde Chivet, Caterina Marchioretti, Marco Pirazzini, et al.
Neuromuscular Disorders : NMD
|
July 23, 2014
Early onset and novel features in a spinal and bulbar muscular atrophy patient with a 68 CAG repeat
Christopher Grunseich, Ilona R Kats, Laura C Bott, et al.
Neurology
|
February 22, 2019
Muscle and not neuronal biomarkers correlate with severity in spinal and bulbar muscular atrophy
Vittoria Lombardi, Giorgia Querin, Oliver J Ziff, et al.
Page
of 7
Search research articles
Search
Showing results (41-50 of 62) with videos related to
Sort By:
Page
of 7
Molecular Therapy. Nucleic Acids
|
February 12, 2021
Targeting the 5' untranslated region of <i>SMN2</i> as a therapeutic strategy for spinal muscular atrophy
Audrey M Winkelsas, Christopher Grunseich, George G Harmison, et al.
EMBO Molecular Medicine
|
September 19, 2023
PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice
Anna J Kordala, Jessica Stoodley, Nina Ahlskog, et al.
Journal of Neurology
|
June 28, 2011
A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results
Francesco Saccà, Mario Quarantelli, Carlo Rinaldi, et al.
Nucleic Acids Research
|
January 23, 2025
Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs
Antonio Garcia-Guerra, Chaitra Sathyaprakash, Olivier G de Jong, et al.
Human Molecular Genetics
|
March 11, 2016
A small-molecule Nrf1 and Nrf2 activator mitigates polyglutamine toxicity in spinal and bulbar muscular atrophy
Laura C Bott, Nisha M Badders, Ke-Lian Chen, et al.
American Journal of Human Genetics
|
December 11, 2012
Cowchock syndrome is associated with a mutation in apoptosis-inducing factor
Carlo Rinaldi, Christopher Grunseich, Irina F Sevrioukova, et al.
Journal of Neurology
|
April 14, 2009
Two novel CYP7B1 mutations in Italian families with SPG5: a clinical and genetic study
Chiara Criscuolo, Alessandro Filla, Giovanni Coppola, et al.
Cells
|
February 6, 2020
Polyglutamine-Expanded Androgen Receptor Alteration of Skeletal Muscle Homeostasis and Myonuclear Aggregation Are Affected by Sex, Age and Muscle Metabolism
Mathilde Chivet, Caterina Marchioretti, Marco Pirazzini, et al.
Neuromuscular Disorders : NMD
|
July 23, 2014
Early onset and novel features in a spinal and bulbar muscular atrophy patient with a 68 CAG repeat
Christopher Grunseich, Ilona R Kats, Laura C Bott, et al.
Neurology
|
February 22, 2019
Muscle and not neuronal biomarkers correlate with severity in spinal and bulbar muscular atrophy
Vittoria Lombardi, Giorgia Querin, Oliver J Ziff, et al.
Page
of 7