dCas9 Fusion Protein With Multiple Effectors for Epigenome Regulation
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Solution Overview
Problem
Current gene editing tools for epigenetic modifications suffer from low transcriptional regulation efficiency and limited scope of modification, particularly in treating diseases caused by epigenetic abnormalities.
Innovation Solution
A fusion is developed comprising a nucleic acid-binding domain and an effector domain, including epigenetic modification and transcriptional regulation factors, which enhances target gene regulation efficiency and modifies the epigenome more effectively by linking different epigenetic modifiers and transcriptional regulation factors.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If current gene editing tools for epigenetic modifications are used, then gene transcription regulation can be achieved, but the transcriptional regulation efficiency is low and the scope of modification is limited
Solution Approach 1:
The patent combines multiple epigenetic regulatory effectors (including DNA methyltransferases, histone modifiers, and transcriptional regulation factors) into a single fusion protein with dCas9. This merging of multiple functional domains into one integrated tool simultaneously improves transcriptional regulation efficiency and expands the scope of epigenetic modification capabilities, resolving the technical contradiction between productivity and adaptability.
Solution Approach 2:
The fusion protein design creates a universal tool that can perform multiple epigenetic modification functions through a single molecule. By incorporating diverse epigenetic regulatory effectors with different functions (methylation, histone modification, transcriptional regulation) into one fusion protein, the system achieves multi-functionality that simultaneously enhances regulation efficiency and broadens modification scope across different target genes.
2Productivity
If multiple epigenetic regulatory effectors are combined into a fusion protein, then transcriptional regulation efficiency and modification scope are improved, but the device complexity increases
Solution Approach 1:
Rather than using multiple separate proteins or complex multi-component systems, the patent merges all epigenetic regulatory effectors into a single fusion protein construct. This consolidation improves regulatory efficiency while managing complexity by integrating multiple functions into one molecular entity with a unified structure that can be expressed and delivered as a single unit.
Data Source
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AI summary
Provided are a fusion and use thereof. The fusion comprises a nucleic acid-binding domain and one or more effector domains; the effector domain comprises one or more of an epigenetic modification domain and/or a transcriptional regulation domain, and the fusion comprises at least two types of epigenetic modification domains and/or transcriptional regulation domains. Also provided is use of the fusion for preparing a product for regulating the expression of a target gene and preparing a related drug.