Phillip Sharp: From RNA Splicing Discovery to RNA-Based Therapeutics
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Lasker Admin
Summary
This article profiles Nobel laureate Phillip Sharp, chronicling his pioneering work in RNA splicing discovery and his transition from basic molecular biology research to founding biotech companies like Biogen and Alnylam. It covers his early career challenges studying complex human cells when bacteria were the preferred model, his landmark discovery of RNA splicing (split genes) in 1977, and his subsequent efforts to translate these fundamental insights into RNA-based therapeutics. The article highlights Sharp's role in advancing RNA interference (RNAi) technology and his perspective on the future of RNA medicines, including the potential of small interfering RNAs (siRNAs) to treat diseases by silencing disease-causing genes.
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Key quotes
· 5 pulledThere was a lot of resistance to the idea of beginning to probe the molecular biology of complex human cells.
The discovery of RNA splicing was a complete surprise. It fundamentally changed our understanding of how genes work.
We realized that if we could understand the basic mechanisms of RNA processing, we could potentially develop entirely new classes of medicines.
RNA interference is one of the most exciting areas in biology today. It gives us the ability to silence any gene in the genome.
The journey from basic discovery to therapeutic application takes decades, but the potential to help patients makes it all worthwhile.
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