Modified RNA G/C Content Jet Injection Expression
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Current RNA-based gene therapy and genetic vaccination methods face challenges due to the instability of mRNA, leading to limited protein expression and the need for repetitive administrations, as well as inefficient delivery of RNA into cells, which hampers therapeutic effectiveness.
Innovation Solution
A modified RNA with increased G/C content in its coding region is administered via jet injection, enhancing protein expression by modifying the G/C content compared to its wild-type counterpart, and utilizing jet injection to improve delivery efficiency.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If RNA is administered by conventional injection methods, then delivery is possible, but delivery efficiency is insufficient
Solution Approach 1:
The patent changes the physical parameters of RNA delivery by modifying G/C content in the coding region, which alters the structural and stability properties of the RNA molecule, thereby improving delivery efficiency and protein expression without changing the amino acid sequence
Solution Approach 2:
The patent performs preliminary modification of the RNA sequence by optimizing G/C content before administration, which pre-enhances the stability and expression characteristics of the RNA, leading to improved therapeutic effectiveness without requiring complex delivery procedures
2Productivity
If unmodified RNA is used, then administration is straightforward, but protein expression is limited and duration is short
Solution Approach 1:
The patent modifies the G/C content parameter in the RNA coding region while maintaining the same amino acid sequence, which enhances protein expression levels and duration without altering the fundamental function or structure of the encoded protein
Solution Approach 2:
The patent applies localized modification only to the coding region's G/C content rather than modifying the entire RNA molecule, which improves expression while maintaining simplicity in other regions of the RNA structure
3Duration of action of moving object
If RNA is administered without G/C content optimization, then sequence is simple, but protein expression duration is limited
Solution Approach 1:
The patent optimizes the G/C content parameter in the coding region to enhance the temporal duration of protein expression, extending the therapeutic effect without requiring complex structural modifications to the RNA molecule
Data Source
Figure 1
Figure 2
Figure 3
AI summary
The invention relates to an RNA comprising at least one open reading frame (ORF) and comprising at least one modification, which increases the expression of the encoded peptide or protein. Furthermore, the invention relates to the medical use of such a modified RNA administered to a subject by jet injection. The invention relates further to a pharmaceutical composition and to a kit of parts comprising said modified RNA for administration by jet injection, preferably for use in the field of gene therapy and/or genetic vaccination. Additionally, the invention relates to a method for enhancing the (localized) expression of RNA-encoded peptides or proteins in the dermis or muscle (of a mammal) comprising administering the modified RNA by jet injection. And finally, the invention relates to a method of treatment comprising administering the modified RNA by jet injection to a subject in need thereof.