Biochemistry of Pre-mRNA Splicing

NIH RePORTER · NIH · R37 · $806,400 · view on reporter.nih.gov ↗

Abstract

The broad objective of this project is to elucidate key mechanistic and regulatory features of human pre-mRNA splicing, and its functional links to nonsense-mediated mRNA decay. Some of these mechanistic insights will be leveraged to develop targeted antisense oligonucleotide therapeutics, which can be used in the long term for clinical applications in precision medicine. The proposed experiments involve a broad range of experimental and computational approaches, including cell-free and cell-based assays, high-throughput RNA-sequencing and quantitative proteomics, bioinformatics, transgenic mouse models, and antisense pharmacology. The anticipated findings will facilitate therapeutic antisense oligonucleotide design for various diseases, besides expanding our fundamental knowledge of basic post-transcriptional mechanisms and regulation.

Key facts

NIH application ID
10197935
Project number
5R37GM042699-31
Recipient
COLD SPRING HARBOR LABORATORY
Principal Investigator
Adrian R Krainer
Activity code
R37
Funding institute
NIH
Fiscal year
2021
Award amount
$806,400
Award type
5
Project period
1989-07-01 → 2023-01-31