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Gene Therapy
|
April 22, 2020
Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
Nina Ahlskog, Daniel Hayler, Anja Krueger, et al.
Turkish Journal of Biology = Turk Biyoloji Dergisi
|
April 30, 2026
Modulating microtubule stability via α-tubulin acetylation partially restores Golgi fragmentation in spinal muscular atrophy
Pelin Zobaroğlu Özer, Memet Gözüböyük, Özge Çetin, et al.
Annals of Neurology
|
August 29, 2012
Glucose metabolism and pancreatic defects in spinal muscular atrophy
Melissa Bowerman, Kathryn J Swoboda, John-Paul Michalski, et al.
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.
Human Molecular Genetics
|
July 27, 2023
AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn2B/- spinal muscular atrophy mice
Sharon J Brown, Darija Šoltić, Silvia A Synowsky, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
September 14, 2016
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
Suzan M Hammond, Gareth Hazell, Fazel Shabanpoor, et al.
JCI Insight
|
July 8, 2021
Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
Katharina E Meijboom, Viola Volpato, Jimena Monzón-Sandoval, et al.
Human Molecular Genetics
|
July 9, 2018
Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice
Lisa M Walter, Christiane E Koch, Corinne A Betts, et al.
Cells
|
November 6, 2020
A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN
Sebastian Rademacher, Nora T Detering, Tobias Schüning, et al.
Neurobiology of Disease
|
June 26, 2017
KCC3 loss-of-function contributes to Andermann syndrome by inducing activity-dependent neuromuscular junction defects
Melissa Bowerman, Céline Salsac, Véronique Bernard, et al.
Page
of 4
Search research articles
Search
Showing results (21-30 of 38) with videos related to
Sort By:
Page
of 4
Gene Therapy
|
April 22, 2020
Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
Nina Ahlskog, Daniel Hayler, Anja Krueger, et al.
Turkish Journal of Biology = Turk Biyoloji Dergisi
|
April 30, 2026
Modulating microtubule stability via α-tubulin acetylation partially restores Golgi fragmentation in spinal muscular atrophy
Pelin Zobaroğlu Özer, Memet Gözüböyük, Özge Çetin, et al.
Annals of Neurology
|
August 29, 2012
Glucose metabolism and pancreatic defects in spinal muscular atrophy
Melissa Bowerman, Kathryn J Swoboda, John-Paul Michalski, et al.
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.
Human Molecular Genetics
|
July 27, 2023
AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn2B/- spinal muscular atrophy mice
Sharon J Brown, Darija Šoltić, Silvia A Synowsky, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
September 14, 2016
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
Suzan M Hammond, Gareth Hazell, Fazel Shabanpoor, et al.
JCI Insight
|
July 8, 2021
Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
Katharina E Meijboom, Viola Volpato, Jimena Monzón-Sandoval, et al.
Human Molecular Genetics
|
July 9, 2018
Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice
Lisa M Walter, Christiane E Koch, Corinne A Betts, et al.
Cells
|
November 6, 2020
A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN
Sebastian Rademacher, Nora T Detering, Tobias Schüning, et al.
Neurobiology of Disease
|
June 26, 2017
KCC3 loss-of-function contributes to Andermann syndrome by inducing activity-dependent neuromuscular junction defects
Melissa Bowerman, Céline Salsac, Véronique Bernard, et al.
Page
of 4