RNA Targeting Compounds with Polymer Backbone for Myotonic Dystrophy
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Solution Overview
Problem
Current methods for studying RNA-ligand interactions are not systematic, limiting the development of compounds that can effectively target RNA motifs for therapeutic applications.
Innovation Solution
Development of RNA targeting compounds with a polymer backbone and pendant RNA binding ligands that can specifically bind to RNA structural and repeat motifs, utilizing peptoid synthesis to vary the spacing and type of RNA binding ligands for enhanced interaction.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Adaptability or versatility
If current methods (SELEX, SAR by MS/NMR, chemical microarrays) are used to study RNA-ligand interactions, then RNA-ligand interactions can be identified, but the study is not systematic and limits rational design
Solution Approach 1:
The patent combines SELEX methodology with chemical microarray technology to create an integrated platform. This merging allows systematic study of RNA-ligand interactions by combining the ligand selection capability of SELEX with the high-throughput screening advantage of chemical microarrays, thereby achieving both systematic analysis and rational design capability
Solution Approach 2:
The developed platform serves multiple functions: it can identify RNA-ligand interactions, characterize binding properties, and facilitate rational design of RNA-targeting compounds. This multi-functionality addresses the limitation of previous methods that could only perform isolated tasks
2Adaptability or versatility
If RNA is used as a drug target, then diverse biological functions can be targeted, but limited information on RNA ligand interactions hinders rational design
Solution Approach 1:
The patent performs preliminary characterization of RNA-ligand interactions using the integrated SELEX-microarray platform before proceeding to rational drug design. This preliminary action generates essential binding information that enables subsequent rational design of RNA-targeting compounds
Solution Approach 2:
The platform provides feedback on ligand binding properties to guide the rational design process. By characterizing RNA-ligand interactions systematically, the method generates information that feeds back into the design of improved ligands, creating a iterative design-improve cycle
3Reliability
If small molecules are designed to target RNA motifs, then therapeutic applications can be achieved, but the lack of systematic identification methods reduces effectiveness
Solution Approach 1:
The patent segments the compound development process into distinct phases: systematic identification of RNA-ligand interactions using the integrated platform, characterization of binding properties, and rational design of therapeutic compounds. This segmentation improves both reliability and productivity by ensuring each phase is systematically addressed
Data Source
AI summary
Disclosed are RNA targeting compounds having the formula:wherein j is an integer from 1 to 100; each i is the same or different and is zero or an integer from 1 to 100; each Z1 represents the same or different linking moiety; each R1 is the same or different and represents an alkyl group or an aryl group; each Q1 represents the same or different RNA binding ligand; Q2 is an alkyl group; Q3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone and two or more pendant RNA binding ligands that are bound to the polymer backbone. Methods for using the subject RNA targeting compounds to treat myotonic dystrophy and other diseases are also disclosed, as are compounds that can be used to prepare the subject RNA targeting compounds.