Genetic Driver Modulation via Non-Canonical Protein Targets
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Solution Overview
Problem
There is a lack of effective therapeutic agents and methods for diagnosis, prevention, and treatment of diseases and conditions with a genetic component, as existing approaches fail to adequately address the role of non-canonical protein targets associated with these conditions.
Innovation Solution
The development of agents that modulate the expression and activity of target proteins identified through non-canonical open reading frames, including polypeptides, polynucleotides, gene editing systems, and cell therapies, to treat and diagnose diseases by targeting specific genetic drivers.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing therapeutic agents and methods are used, then treatment of diseases with genetic component is attempted, but they fail to adequately address non-canonical protein targets
Solution Approach 1:
The patent segments the genetic code analysis into canonical and non-canonical ORF components, allowing separate identification and targeting of previously overlooked non-canonical protein targets while maintaining existing approaches for canonical targets
Solution Approach 2:
The patent develops a universal framework for identifying and targeting both canonical and non-canonical protein drivers across multiple disease types, making the therapeutic approach adaptable to various genetic conditions while maintaining core functionality
2Adaptability or versatility
If non-canonical ORFs are targeted, then new therapeutic opportunities are created, but complexity of identification and validation increases
Solution Approach 1:
The patent performs preliminary computational screening and filtering of non-canonical ORFs using established criteria before experimental validation, reducing the complexity of subsequent identification and validation steps by pre-processing the genetic data
Solution Approach 2:
The patent introduces computational algorithms and bioinformatics tools as intermediaries between raw genomic data and experimental validation, simplifying the identification process by mediating the analysis of non-canonical ORFs through structured computational pipelines
3Quantity of substance
If comprehensive genetic screening is performed, then more genetic drivers are identified, but time and resources required increase
Solution Approach 1:
The patent applies partial screening approaches by focusing on specific genomic regions or disease-associated variants rather than exhaustive whole-genome analysis, identifying sufficient genetic drivers for therapeutic targeting without requiring complete comprehensive screening
Solution Approach 2:
The patent enhances screening resolution by applying different analysis strategies to different genomic regions, using more rigorous analysis for high-priority areas and streamlined analysis for lower-priority regions, optimizing the balance between identification completeness and resource utilization
Data Source
AI summary
The disclosure provides, in various embodiments, compositions, such as polypeptides, polynucleotides, gene editing systems, small molecules, vectors or host cells, that comprise and/or modulate expression or activity of immune regulation-associated proteins, such as cytokines. The disclosure also provides, in various embodiments, methods of treating a disease or condition (e.g., a disease or condition associated with the Genome-Wide Association Study (GWAS), the Cancer Genome Atlas (TCGA), whole genome sequencing, phenome-wide association study (PheWAS), expression quantitative trait locus (cQTL) studies, or a combination thereof) using an agent that comprises and/or modulates expression or activity of an immune regulation-associated protein, and methods of identifying said agent.