Recombinant Vector CTE Segmentation for Stable Multi-Gene Expression
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Solution Overview
Problem
Current gene therapy technologies face challenges in delivering and stably expressing multiple target genes in cells, particularly due to limitations in vector size, gene silencing issues, and difficulties in predicting or preventing non-uniform silencing of multiple genes.
Innovation Solution
A recombinant vector is designed with a constitutive transport element (CTE), two or more target genes, and corresponding promoters, where the first target gene and its promoter are in reverse orientation, enabling simultaneous expression of multiple genes from a single vector.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If multiple target genes are contained in a single vector, then the gene delivery efficiency is improved, but the possibility of non-uniform silencing increases
Solution Approach 1:
The patent divides the vector into multiple independent expression units, each containing a target gene and its corresponding promoter. This segmentation allows each gene to be expressed independently, preventing silencing of individual genes when multiple genes are present in the same vector. The constitutive transport element (CTE) is shared among all genes, enabling efficient delivery while maintaining uniform expression stability.
2Quantity of substance
If the size of the target gene increases, then the gene delivery capacity is improved, but the possibility of gene loss due to homologous recombination increases
Solution Approach 1:
The patent segments large genes into multiple smaller expression units, each with its own promoter. This reduces the size of individual genetic elements that could undergo homologous recombination, thereby maintaining gene stability while still delivering multiple genes through the shared CTE framework.
3Productivity
If viral vectors are used for gene delivery, then the gene transfer efficiency is improved, but the induction of immune response and toxicity increases
Solution Approach 1:
The patent uses a constitutive transport element (CTE) as an intermediary component that enables efficient gene delivery without triggering immune responses. The CTE acts as a non-viral delivery mechanism that maintains high gene transfer efficiency while avoiding the harmful effects associated with viral vectors, such as immune response and toxicity.
Data Source
AI summary
The present invention relates to a recombinant vector, and a gene delivery system, recombinant virus and transformant comprising the same. The recombinant vector according to the present invention is designed to express one target gene by one promoter, and is capable of delivering two or more target genes into cells and stably expressing up to three target genes simultaneously from a single vector. Thus, this recombinant vector may be useful as an efficient gene delivery system.