Enhanced Self-Replicating Cell Selective Gene Delivery Compositions and Methods
Legal Citation
Summary of the Inventive Concept
Improved gene delivery compositions and methods that enable targeted, efficient, and safe gene expression in specific cell types, overcoming limitations of existing self-replicating polyribonucleotide-based approaches.
Background and Problem Solved
Despite the progress in gene therapy, existing self-replicating polyribonucleotide-based approaches suffer from limitations, including off-target effects, inefficient gene expression, and lack of cell-type specificity. The new inventive concept addresses these limitations by incorporating cell-type specific promoters, receptors, and microRNA target sequences, ensuring targeted gene delivery and reducing off-target effects.
Detailed Description of the Inventive Concept
The enhanced self-replicating polyribonucleotide compositions comprise cell-type specific promoters, receptors, and microRNA target sequences, enabling targeted uptake by specific cell types. The polyribonucleotides can be administered via various delivery vehicles, including viral vectors, lipids, and nanoparticles. The inventive concept also encompasses methods for delivering genes to specific cell types, enhancing gene expression, and treating diseases using these compositions.
Novelty and Inventive Step
The new claims introduce novel features, including cell-type specific promoters, receptors, and microRNA target sequences, which significantly improve the specificity, efficiency, and safety of gene delivery. These features are not obvious in light of the original patent and represent a significant advancement in the field.
Alternative Embodiments and Variations
Alternative embodiments of the inventive concept include using different types of promoters, receptors, and microRNA target sequences, as well as combining these elements in various ways to achieve targeted gene delivery. Variations of the inventive concept could also include using different delivery vehicles, such as electroporation or sonoporation, to enhance gene expression.
Potential Commercial Applications and Market
The enhanced self-replicating polyribonucleotide compositions and methods have significant commercial potential in the gene therapy market, particularly for treating genetic disorders, cancer, and infectious diseases. The targeted and efficient gene delivery enabled by this inventive concept could lead to improved treatment outcomes, reduced side effects, and increased patient safety.
CPC Classifications
| Section | Class | Group |
|---|---|---|
| A | A61 | A61K38/1709 |
| A | A61 | A61K31/7088 |
| A | A61 | A61K31/7115 |
| C | C12 | C12Y207/07048 |
| C | C12 | C12N2310/113 |
| C | C12 | C12N2710/10343 |
Section 103 Obviousness Analysis (PHOSITA)
Field of Art
Molecular biology and gene therapy, specifically focusing on self-replicating RNA-based gene delivery systems, with expertise in viral vector design, RNA modification, and targeted cellular gene expression
Person of Ordinary Skill (PHOSITA) Profile
A molecular biologist or genetic engineer with advanced degree, experienced in RNA engineering, viral vector design, and gene delivery techniques, familiar with cell-type specific targeting strategies and RNA modification technologies
Obviousness Rationale
A person of ordinary skill would recognize that the PTD's variations represent predictable extensions of the source patent's core technology by applying known strategies for enhancing gene delivery specificity and efficiency. The proposed modifications involve standard techniques in RNA engineering and cellular targeting that would be well within the capabilities of a skilled practitioner. The combination of cell-type specific promoters, microRNA targeting, and refined delivery mechanisms represents an incremental improvement using conventional molecular biology approaches.
Obvious Combinations & Variations
Original Patent Information
| Patent Number | US 11,857,598 |
|---|---|
| Title | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| Assignee(s) | University of South Florida |