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Molecular Therapy. Methods & Clinical Development
|
November 11, 2024
An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Vidyullatha Vasireddy, Casey A Maguire, David W Anderson, et al.
The Journal of Clinical Investigation
|
March 23, 2023
Sphingolipid desaturase DEGS1 is essential for mitochondria-associated membrane integrity
Laura Planas-Serra, Nathalie Launay, Leire Goicoechea, et al.
Neurogenetics
|
January 16, 2020
POLR3A variants with striatal involvement and extrapyramidal movement disorder
Inga Harting, Murtadha Al-Saady, Ingeborg Krägeloh-Mann, et al.
Cell Metabolism
|
May 13, 2014
Pharmacological Inhibition of poly(ADP-ribose) polymerases improves fitness and mitochondrial function in skeletal muscle
Eija Pirinen, Carles Cantó, Young Suk Jo, et al.
Clinical Pharmacology and Therapeutics
|
July 25, 2017
Genetic Variants in HSD17B3, SMAD3, and IPO11 Impact Circulating Lipids in Response to Fenofibrate in Individuals With Type 2 Diabetes
Daniel M Rotroff, Sonja S Pijut, Skylar W Marvel, et al.
The Journal of Molecular Diagnostics : JMD
|
May 15, 2022
Systematic Collaborative Reanalysis of Genomic Data Improves Diagnostic Yield in Neurologic Rare Diseases
Gemma Bullich, Leslie Matalonga, Montserrat Pujadas, et al.
Clinical Genetics
|
April 27, 2020
HNRNPH1-related syndromic intellectual disability: Seven additional cases suggestive of a distinct syndromic neurodevelopmental syndrome
Sara C Reichert, Rachel Li, Scott A Turner, et al.
European Journal of Neurology
|
August 14, 2023
Frequency and phenotypic spectrum of spinocerebellar ataxia 27B and other genetic ataxias in a Spanish cohort of late-onset cerebellar ataxia
Pablo Iruzubieta, David Pellerin, Alberto Bergareche, et al.
Eclinicalmedicine
|
June 30, 2022
Epigenetic profiling linked to multisystem inflammatory syndrome in children (MIS-C): A multicenter, retrospective study
Veronica Davalos, Carlos A García-Prieto, Gerardo Ferrer, et al.
Ebiomedicine
|
September 8, 2023
Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy
Isabelle Weinhofer, Paulus Rommer, Andreas Gleiss, et al.
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of 14
Search research articles
Search
Showing results (91-100 of 135) with videos related to
Sort By:
Page
of 14
Molecular Therapy. Methods & Clinical Development
|
November 11, 2024
An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Vidyullatha Vasireddy, Casey A Maguire, David W Anderson, et al.
The Journal of Clinical Investigation
|
March 23, 2023
Sphingolipid desaturase DEGS1 is essential for mitochondria-associated membrane integrity
Laura Planas-Serra, Nathalie Launay, Leire Goicoechea, et al.
Neurogenetics
|
January 16, 2020
POLR3A variants with striatal involvement and extrapyramidal movement disorder
Inga Harting, Murtadha Al-Saady, Ingeborg Krägeloh-Mann, et al.
Cell Metabolism
|
May 13, 2014
Pharmacological Inhibition of poly(ADP-ribose) polymerases improves fitness and mitochondrial function in skeletal muscle
Eija Pirinen, Carles Cantó, Young Suk Jo, et al.
Clinical Pharmacology and Therapeutics
|
July 25, 2017
Genetic Variants in HSD17B3, SMAD3, and IPO11 Impact Circulating Lipids in Response to Fenofibrate in Individuals With Type 2 Diabetes
Daniel M Rotroff, Sonja S Pijut, Skylar W Marvel, et al.
The Journal of Molecular Diagnostics : JMD
|
May 15, 2022
Systematic Collaborative Reanalysis of Genomic Data Improves Diagnostic Yield in Neurologic Rare Diseases
Gemma Bullich, Leslie Matalonga, Montserrat Pujadas, et al.
Clinical Genetics
|
April 27, 2020
HNRNPH1-related syndromic intellectual disability: Seven additional cases suggestive of a distinct syndromic neurodevelopmental syndrome
Sara C Reichert, Rachel Li, Scott A Turner, et al.
European Journal of Neurology
|
August 14, 2023
Frequency and phenotypic spectrum of spinocerebellar ataxia 27B and other genetic ataxias in a Spanish cohort of late-onset cerebellar ataxia
Pablo Iruzubieta, David Pellerin, Alberto Bergareche, et al.
Eclinicalmedicine
|
June 30, 2022
Epigenetic profiling linked to multisystem inflammatory syndrome in children (MIS-C): A multicenter, retrospective study
Veronica Davalos, Carlos A García-Prieto, Gerardo Ferrer, et al.
Ebiomedicine
|
September 8, 2023
Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy
Isabelle Weinhofer, Paulus Rommer, Andreas Gleiss, et al.
Page
of 14