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RNA (New York, N.Y.)
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January 19, 2017
The nucleoside analog clitocine is a potent and efficacious readthrough agent
Westley J Friesen, Christopher R Trotta, Yuki Tomizawa, et al.
American Journal of Human Genetics
|
March 26, 2019
ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia
Elisabetta Morini, Dadi Gao, Connor M Montgomery, et al.
Human Molecular Genetics
|
March 3, 2016
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy
Xin Zhao, Zhihua Feng, Karen K Y Ling, et al.
Bioorganic & Medicinal Chemistry Letters
|
October 17, 2017
4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis
Michael A Arnold, Aleksey I Gerasyuto, Jiashi Wang, et al.
Human Molecular Genetics
|
October 11, 2023
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models
Grace Liu, Philip Lipari, Anna Mollin, et al.
American Journal of Human Genetics
|
February 22, 2023
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
Elisabetta Morini, Anil Chekuri, Emily M Logan, et al.
Blood Advances
|
January 26, 2021
DHODH inhibition synergizes with DNA-demethylating agents in the treatment of myelodysplastic syndromes
Kensuke Kayamori, Yurie Nagai, Cheng Zhong, et al.
Journal of Medicinal Chemistry
|
June 15, 2016
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
Matthew G Woll, Hongyan Qi, Anthony Turpoff, et al.
Bioorganic & Medicinal Chemistry Letters
|
May 21, 2013
Discovery of novel HCV inhibitors: synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B
Xiaoyan Zhang, Nanjing Zhang, Guangming Chen, et al.
Molecular Cancer Therapeutics
|
July 28, 2021
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent
Finith Jernigan, Arthur Branstrom, John D Baird, et al.
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of 7
Search research articles
Search
Showing results (41-50 of 67) with videos related to
Sort By:
Page
of 7
RNA (New York, N.Y.)
|
January 19, 2017
The nucleoside analog clitocine is a potent and efficacious readthrough agent
Westley J Friesen, Christopher R Trotta, Yuki Tomizawa, et al.
American Journal of Human Genetics
|
March 26, 2019
ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia
Elisabetta Morini, Dadi Gao, Connor M Montgomery, et al.
Human Molecular Genetics
|
March 3, 2016
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy
Xin Zhao, Zhihua Feng, Karen K Y Ling, et al.
Bioorganic & Medicinal Chemistry Letters
|
October 17, 2017
4-Hydroxy-2-pyridones: Discovery and evaluation of a novel class of antibacterial agents targeting DNA synthesis
Michael A Arnold, Aleksey I Gerasyuto, Jiashi Wang, et al.
Human Molecular Genetics
|
October 11, 2023
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models
Grace Liu, Philip Lipari, Anna Mollin, et al.
American Journal of Human Genetics
|
February 22, 2023
Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia
Elisabetta Morini, Anil Chekuri, Emily M Logan, et al.
Blood Advances
|
January 26, 2021
DHODH inhibition synergizes with DNA-demethylating agents in the treatment of myelodysplastic syndromes
Kensuke Kayamori, Yurie Nagai, Cheng Zhong, et al.
Journal of Medicinal Chemistry
|
June 15, 2016
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
Matthew G Woll, Hongyan Qi, Anthony Turpoff, et al.
Bioorganic & Medicinal Chemistry Letters
|
May 21, 2013
Discovery of novel HCV inhibitors: synthesis and biological activity of 6-(indol-2-yl)pyridine-3-sulfonamides targeting hepatitis C virus NS4B
Xiaoyan Zhang, Nanjing Zhang, Guangming Chen, et al.
Molecular Cancer Therapeutics
|
July 28, 2021
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent
Finith Jernigan, Arthur Branstrom, John D Baird, et al.
Page
of 7