mdl-201053 and Muscular-Atrophy--Spinal

mdl-201053 has been researched along with Muscular-Atrophy--Spinal* in 1 studies

Other Studies

1 other study(ies) available for mdl-201053 and Muscular-Atrophy--Spinal

Article	Year
Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology. Life science alliance, 2019, Volume: 2, Issue:2 Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is caused by reduced levels of functional survival motor neuron (SMN) protein. To identify therapeutic agents for SMA, we established a versatile SMN2-GFP reporter line by targeting the human Topics: Animals; Blood-Brain Barrier; Cysteine Proteinase Inhibitors; Dipeptides; Disease Models, Animal; Drug Evaluation, Preclinical; Genes, Reporter; HEK293 Cells; Humans; Ketones; Leucine; Mice; Mitochondria; Motor Neurons; Muscular Atrophy, Spinal; Protective Agents; Protein Stability; Proteolysis; Signal Transduction; Survival of Motor Neuron 2 Protein; Transfection; Treatment Outcome	2019

Article

Year

Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology.

Life science alliance, 2019, Volume: 2, Issue:2

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is caused by reduced levels of functional survival motor neuron (SMN) protein. To identify therapeutic agents for SMA, we established a versatile SMN2-GFP reporter line by targeting the human

Topics: Animals; Blood-Brain Barrier; Cysteine Proteinase Inhibitors; Dipeptides; Disease Models, Animal; Drug Evaluation, Preclinical; Genes, Reporter; HEK293 Cells; Humans; Ketones; Leucine; Mice; Mitochondria; Motor Neurons; Muscular Atrophy, Spinal; Protective Agents; Protein Stability; Proteolysis; Signal Transduction; Survival of Motor Neuron 2 Protein; Transfection; Treatment Outcome

2019