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Novel RNA Drug Discovery Tool Overcomes Undruggable Targets
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December 16, 2020
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Despite the scientific advancements made in creating medicines that can target and treat diseases, researchers still face the challenge of targeting “undruggable” targets. Most drugs target proteins to treat diseases, however, there are some proteins that conventional drugs cannot access for reasons such as a protein molecule’s shape or how it folds. There have been many strategies and tools developed to overcome these challenges such as targeting RNA. RNAs have not been viewed as drug targets as of recent due to their short-lived existence, changeable shape, and limited array of building blocks. Now, researchers at Scripps Research report they have developed a new RNA drug discovery tool that allows rapid drug discovery and optimization of RNA-targeting compounds.
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IMAGE: Scripps Research Chemist Matthew Disney, PhD, and graduate student Blessy Suresh in their Jupiter, Florida lab. view more
Credit: Scripps Research
JUPITER, Fla. Dec. 14, 2020 Imagine trying to throw a bullseye when the dartboard lies buried within a crumpled box. That s the challenge faced by scientists working to make new medicines for some undruggable diseases, including a type of metastatic breast cancer.
Scientists refer to diseases as undruggable when their targets lie on a protein molecule that folds inward, or in a way that shields the active site from would-be treatments.
Addressing this problem of undruggable proteins, a team of Scripps Research scientists has invented a tool that bypasses the awkward proteins completely, and instead modifies elements involved in their construction and regulation. This new tool, called Chem-CLIP-Fragment Mapping, focuses on RNA, molecules that read genes and help build proteins, among other duties.
New tool aids in the discovery of RNA medicines for undruggable diseases
Imagine trying to throw a bullseye when the dartboard lies buried within a crumpled box. That s the challenge faced by scientists working to make new medicines for some undruggable diseases, including a type of metastatic breast cancer.
Scientists refer to diseases as undruggable when their targets lie on a protein molecule that folds inward, or in a way that shields the active site from would-be treatments.
Addressing this problem of undruggable proteins, a team of Scripps Research scientists has invented a tool that bypasses the awkward proteins completely, and instead modifies elements involved in their construction and regulation. This new tool, called Chem-CLIP-Fragment Mapping, focuses on RNA, molecules that read genes and help build proteins, among other duties.