Nucleic Acid Construct for Selective mRNA Cleavage
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Solution Overview
Problem
Current genome editing and RNA editing technologies face challenges such as low selectivity and versatility in targeting therapeutic genes, leading to significant side effects and resistance issues in cancer and infectious disease treatments.
Innovation Solution
A nucleic acid construct comprising a guide RNA portion that binds to target RNAs and an RNA-cleaving Cas protein expression portion, specifically designed to target mutations in vertebrate cells, viruses, or bacteria, allowing for precise RNA cleavage and reduced expression in cancer cells or infected cells without affecting normal cells.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If genome editing or RNA editing is used to regulate gene expression, then therapeutic effects can be achieved, but selectivity for target genes is low and serious side effects occur
Solution Approach 1:
The patent applies local quality by designing guide RNA with a specific structure that includes a scaffold region and a target-binding region. The target-binding region is specifically engineered to hybridize with complementary sequences in the target gene, enabling selective recognition and regulation of specific genes while leaving other genes unaffected. This localized specificity resolves the contradiction between achieving therapeutic effects and avoiding side effects.
2Adaptability or versatility
If siRNA or shRNA is used to regulate mRNA expression, then gene expression can be suppressed, but versatility in affecting therapeutic targets is insufficient
Solution Approach 1:
The patent implements universality by creating a modular RNA interference system where the guide RNA can be easily reconfigured to target different genes. The standardized scaffold structure combined with customizable target-binding regions allows the same basic mechanism to be applied across multiple therapeutic targets, achieving both versatility in target selection and maintained selectivity through the consistent use of specific hybridization-based recognition.
3Reliability
If antiviral agents or antibacterial agents are used to treat infectious diseases, then therapeutic effects are achieved, but resistance develops
Solution Approach 1:
The patent applies feedback by designing the RNA interference system to continuously regulate target gene expression based on the presence of viral or bacterial RNA. The guide RNA directs the RNA-induced silencing complex to specifically bind and cleave target mRNA, creating a self-regulating system that adapts to the presence of pathogens. This mechanism reduces the likelihood of resistance development compared to conventional agents that exert continuous selective pressure.
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
The nucleic acid construct enhances specificity and potency in treating cancer and infectious diseases by selectively reducing mRNA expression in targeted cells, minimizing side effects and overcoming resistance, while being compatible with conventional therapeutic methods.
Implementation Method 1
at least one guide RNA portion that binds to one or more target RNAs
Implementation Method 2
an RNA-cleaving Cas protein expression portion
Data Source
AI summary
The present invention provides a nucleic acid construct containing at least one guide RNA portion that binds to one or more target RNAs and an RNA-cleaving Cas protein expression portion, wherein the one or more target RNAs are derived from a mutation in a vertebrate cell, a virus, or a bacterium.