Genetically Encoded, Biologically Responsive, And Programmable Bispecific Aptames As Therapeutic Protein Degraders

Patent Status

Patent Pending

Brief Description

Engineered ribonucleic acid aptamers are designed with a modular architecture to target and bind specific proteins. Developed by researchers at UC Berkeley, these molecular tools include a domain A serving as a first target protein binding domain, a domain B functioning as a linker, and a domain C acting as a second target protein binding domain. In certain configurations, the aptamers can incorporate a domain D that undergoes a distinct conformational change when engaged by a specific ligand, enabling precise control over their activity.

Suggested uses

• Development of targeted therapeutics that direct specific disease causing proteins to cellular degradation machinery

• Genomic engineering applications utilizing donor deoxyribonucleic acid to integrate programmable regulatory sequences into safe harbor loci

• High throughput screening assays to identify novel ribonucleic acid aptamers and functional protein binding domains

• Creation of ligand responsive molecular switches for real time control of gene expression in synthetic biology frameworks

• Diagnostic tools configured to detect and bind specific target proteins within complex biological samples

Advantages

• Offers a highly modular and programmable architecture with multiple distinct domains for multifunctional protein targeting

• Enables precise external control over molecular interactions through ligand induced conformational changes in specific domains

• Provides enhanced molecular stability and diverse structural options through circularization and flanking ribozyme sequences

• Allows for stable and predictable genomic integration by utilizing donor deoxyribonucleic acid targeted to safe harbor loci

• Streamlines the discovery of new targeting tools via integrated methods for screening diverse aptamer sequences

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