RNA-Targeting Splicing Modifiers: Drug Development and Screening Assays

• Published in: Molecules (Basel, Switzerland) Vol. 26; no. 8; p. 2263
• Main Authors: Tang, Zhichao, Zhao, Junxing, Pearson, Zach J., Boskovic, Zarko V., Wang, Jingxin
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 14.04.2021; MDPI
• Subjects: Adenosine; alternative splicing; Animals; antisense oligonucleotide; Bacteria; Base Sequence; Chloroplasts; Disease; Disease - genetics; Drug Development; Drug Evaluation, Preclinical; Genes; high-throughput screening; Humans; Mitochondria; Mutation; Neuromuscular diseases; Proteins; Review; RNA Splicing - genetics; RNA-targeting; small molecule; Small Molecule Libraries - pharmacology; Spliceosomes - metabolism; splicing modifier; Transfer RNA; splicing modifier; small molecule; alternative splicing; antisense oligonucleotide; high-throughput screening; RNA-targeting

RNA splicing is an essential step in producing mature messenger RNA (mRNA) and other RNA species. Harnessing RNA splicing modifiers as a new pharmacological modality is promising for the treatment of diseases caused by aberrant splicing. This drug modality can be used for infectious diseases by disr...