Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy. [electronic resource]
- Journal of medicinal chemistry Feb 2008
- 449-69 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
0022-2623
10.1021/jm061475p doi
Aminoquinolines--chemical synthesis Animals Biological Availability Blood-Brain Barrier--metabolism Cell Line Cells, Cultured Cyclic AMP Response Element-Binding Protein--genetics Fibroblasts--drug effects Folic Acid Antagonists--chemical synthesis Heterozygote Humans Mice Models, Molecular Molecular Conformation Muscular Atrophy, Spinal--drug therapy Nerve Tissue Proteins--genetics Permeability Piperidines--chemical synthesis Promoter Regions, Genetic Quinazolines--chemical synthesis RNA-Binding Proteins--genetics SMN Complex Proteins Spinal Muscular Atrophies of Childhood--genetics Stereoisomerism Structure-Activity Relationship Survival of Motor Neuron 1 Protein Survival of Motor Neuron 2 Protein Tetrahydrofolate Dehydrogenase--chemistry