Gene Therapy Vector VTvafl7 for Enhanced Cellular Penetration
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Solution Overview
Problem
Current gene therapy vectors, particularly plasmid vectors, face limitations such as the presence of antibiotic resistance genes, viral genome sequences, and excessive length, which hinder their efficiency and safety for human and animal use, especially in terms of penetration into eukaryotic cells and potential immune responses.
Innovation Solution
Development of gene therapy DNA vectors like VTvafl7 that exclude antibiotic resistance genes and viral genome sequences, optimized for smaller size to enhance penetration and expression of therapeutic genes NOS2, NOS3, VIP, KCNMA1, and CGRP, using specific restriction sites and oligonucleotides for cloning, and produced through antibiotic-free media to ensure safety and efficacy.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional plasmid vectors are used for gene therapy, then gene delivery can be achieved, but the vectors contain antibiotic resistance genes and viral genome sequences that cause immune responses and reduce safety
Solution Approach 1:
The patent extracts and removes harmful components (antibiotic resistance genes and viral genome sequences) from the plasmid vector structure. The resulting vector contains only essential elements: origin of replication, promoter, and therapeutic gene, eliminating immunogenic elements while maintaining gene delivery functionality.
2Reliability
If conventional plasmid vectors with full regulatory elements are used, then gene expression can be maintained, but the vector length becomes excessive which hinders penetration into eukaryotic cells
Solution Approach 1:
The patent extracts only the essential regulatory elements (origin of replication and promoter) while removing non-essential sequences. This minimizes vector length to enhance cellular uptake efficiency while preserving core functions of replication and gene expression.
Solution Approach 2:
The patent changes the structural parameters of the vector by reducing DNA length and optimizing the arrangement of essential elements. This parameter optimization improves physical properties relevant to cellular penetration while maintaining biological functionality.
3Ease of manufacture
If antibiotic resistance genes are included in the vector for selection purposes, then production and selection of transformed cells is easier, but the vectors become unsafe for human and animal use
Solution Approach 1:
The patent removes antibiotic resistance genes from the vector structure, eliminating the safety hazard while maintaining the ability to produce and select transformed cells through alternative methods that do not require antibiotic markers.
Data Source
AI summary
The invention refers to genetic engineering and can be used in biotechnology, medicine, and agriculture for the manufacture of gene therapy products. Gene therapy DNA vector based on the gene therapy DNA vector VTvaflV carrying the therapeutic gene selected from the group of NOS2, NOS3, VIP, KCNMA1, and CGRP genes is constructed in order to increase the expression level of this therapeutic gene in humans and animals, while gene therapy DNA vector VTvafl7-NOS2, or VTvaflV-NOS3, or VTvafl7-VIP, or VTvafl7-KCNMAI, or VTvafl7-CGRP has the nucleotide sequence SEQ ID No. 1, or SEQ ID No. 2, or SEQ ID No. 3, or SEQ ID No. 4, or SEQ ID No. 5, respectively.