Compact Gene Therapy DNA Vector VTvaf17 for Enhanced Cellular Penetration
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Solution Overview
Problem
Current gene therapy DNA vectors for increasing the expression of SHH, CTNNB1, NOG, and WNT7A genes face challenges due to the presence of antibiotic resistance genes, viral regulatory elements, and excessive length, which can lead to safety concerns and reduced efficacy in penetrating eukaryotic cells.
Innovation Solution
Development of gene therapy DNA vectors, such as VTvaf17, that are designed to be free of antibiotic resistance genes and viral regulatory elements, with a compact size to enhance penetration and expression efficiency, using specific restriction sites and oligonucleotides for cloning, and produced using antibiotic-free media to ensure safety and industrial scalability.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If traditional gene therapy DNA vectors are used, then they can carry therapeutic genes, but they contain antibiotic resistance genes and viral regulatory elements that cause safety concerns
Solution Approach 1:
The patent removes antibiotic resistance genes and viral regulatory elements from the DNA vector structure, extracting only the essential functional components needed for gene delivery and expression while eliminating harmful elements
2Reliability
If traditional DNA vectors are used, then they can express therapeutic genes, but their excessive length reduces penetration efficiency into eukaryotic cells
Solution Approach 1:
The patent extracts and removes non-essential sequences from the vector structure, including excessive regulatory elements and antibiotic resistance genes, to reduce the overall vector length to an optimal range for cellular penetration while preserving core functionality
Solution Approach 2:
The patent optimizes the vector length parameter by removing unnecessary sequences, transforming the vector from an excessively long structure to a compact form that maintains gene expression capability while achieving efficient cellular uptake
3Ease of manufacture
If vectors with antibiotic resistance genes are used for production, then they are easier to select and propagate, but they require antibiotic-containing media that complicates industrial production and raises safety concerns
Solution Approach 1:
The patent removes antibiotic resistance genes from the vector system entirely, eliminating the dependency on antibiotic-containing media for bacterial selection and propagation, thereby enabling antibiotic-free industrial production
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. A gene therapy DNA vector based on the VTvaf17 gene therapy DNA vector is proposed that carries a target gene selected from the group of SHH, CTNNB1, NOG, WNT7A genes for the treatment of diseases characterized by impaired tissue regeneration, wound healing, growth, pigmentation and hair coloring, formation and maturation of hair follicles, processes of differentiation and growth of cells, leading to a decrease in the activity of hair follicles, including with allopecia, autoimmune diseases, hereditary and acquired pathological conditions thawing, and for accelerated healing of wounds, restoration of the hairline and the prevention and inhibition of alopecia. Moreover, the gene therapy DNA vector VTvaf17-SHH, or VTvaf 17 -CTNNB 1, or VTvaf17-NOG, or VTvaf17-WNT7A has the nucleotide sequence of SEQ ID No. 1 or SEQ ID No. 2 or SEQ ID No. 3 or SEQ ID No. 4, respectively. Also provided are a method of producing said vector, the use of a vector, a strain of Escherichia coli carrying said vector, as well as a method of industrial production of said vector.