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Science Translational Medicine
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April 22, 2026
Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i>-related neurodevelopmental disorder
Ane Korff, Xiaojing Yang, Ozan Ozdemir, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
March 9, 2017
SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage
Mohini Jangi, Christina Fleet, Patrick Cullen, et al.
Brain : a Journal of Neurology
|
July 3, 2018
CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis
Eva Janzen, Natalia Mendoza-Ferreira, Seyyedmohsen Hosseinibarkooie, et al.
Biorxiv : the Preprint Server for Biology
|
November 24, 2025
Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i>-related neurodevelopmental disorder
Ane Korff, Xiaojing Yang, Ozan Ozdemir, et al.
Science Advances
|
August 22, 2025
An alternative cytoplasmic SFPQ isoform with reduced phase separation potential is up-regulated in ALS
Jacob Neeves, Marija Petrić Howe, Oliver J Ziff, et al.
Nature Neuroscience
|
June 4, 2021
Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
Roy Maimon, Carlos Chillon-Marinas, Cedric E Snethlage, et al.
The Journal of Pharmacology and Experimental Therapeutics
|
May 3, 2014
Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
Frank Rigo, Seung J Chun, Daniel A Norris, et al.
Journal of Medicinal Chemistry
|
April 14, 2006
Evaluation of basic amphipathic peptides for cellular delivery of antisense peptide nucleic acids
Martin A Maier, Christine C Esau, Andrew M Siwkowski, et al.
Nucleic Acids Research
|
December 4, 2020
High-resolution visualization and quantification of nucleic acid-based therapeutics in cells and tissues using Nanoscale secondary ion mass spectrometry (NanoSIMS)
Cuiwen He, Michael T Migawa, Kai Chen, et al.
Neuron
|
December 27, 2016
The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
Constantin d'Ydewalle, Daniel M Ramos, Noah J Pyles, et al.
Page
of 17
Search research articles
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Showing results (91-100 of 169) with videos related to
Sort By:
Page
of 17
Science Translational Medicine
|
April 22, 2026
Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i>-related neurodevelopmental disorder
Ane Korff, Xiaojing Yang, Ozan Ozdemir, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
March 9, 2017
SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage
Mohini Jangi, Christina Fleet, Patrick Cullen, et al.
Brain : a Journal of Neurology
|
July 3, 2018
CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis
Eva Janzen, Natalia Mendoza-Ferreira, Seyyedmohsen Hosseinibarkooie, et al.
Biorxiv : the Preprint Server for Biology
|
November 24, 2025
Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i>-related neurodevelopmental disorder
Ane Korff, Xiaojing Yang, Ozan Ozdemir, et al.
Science Advances
|
August 22, 2025
An alternative cytoplasmic SFPQ isoform with reduced phase separation potential is up-regulated in ALS
Jacob Neeves, Marija Petrić Howe, Oliver J Ziff, et al.
Nature Neuroscience
|
June 4, 2021
Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
Roy Maimon, Carlos Chillon-Marinas, Cedric E Snethlage, et al.
The Journal of Pharmacology and Experimental Therapeutics
|
May 3, 2014
Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
Frank Rigo, Seung J Chun, Daniel A Norris, et al.
Journal of Medicinal Chemistry
|
April 14, 2006
Evaluation of basic amphipathic peptides for cellular delivery of antisense peptide nucleic acids
Martin A Maier, Christine C Esau, Andrew M Siwkowski, et al.
Nucleic Acids Research
|
December 4, 2020
High-resolution visualization and quantification of nucleic acid-based therapeutics in cells and tissues using Nanoscale secondary ion mass spectrometry (NanoSIMS)
Cuiwen He, Michael T Migawa, Kai Chen, et al.
Neuron
|
December 27, 2016
The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
Constantin d'Ydewalle, Daniel M Ramos, Noah J Pyles, et al.
Page
of 17