Nucleic Acid –Targeted Small Molecules have Therapeutic Potential in the Treatment of Spinal Muscular Atrophy: Small‐molecule drugs that can selectively bind RNA and modulate pre‐mRNA splicing have potential as a treatment strategy for human disease, including spinal muscular atrophy

American Journal of Medical Genetics Part A,Volume 176, Issue 8, Page 1698-1699, August 2018.
Source: American Journal of Medical Genetics Part A - Category: Genetics & Stem Cells Source Type: research