Enhanced Rod Photoreceptor-Specific Gene Expression System
Legal Citation
Summary of the Inventive Concept
An optimized gene expression system for specific targeting of rod photoreceptors, offering improved efficiency, safety, and versatility.
Background and Problem Solved
The original SYNP161 promoter-based expression cassette, while effective, has limitations in terms of transcriptional activity and specificity. The new inventive concept addresses these limitations by introducing enhanced nucleic acid sequences and regulatory elements, ensuring more efficient and targeted gene expression in rod photoreceptors.
Detailed Description of the Inventive Concept
The enhanced system comprises a nucleic acid sequence of at least 150 base pairs with at least 97% identity to SEQ ID NO:1, specifically designed to drive gene expression in rod photoreceptors. The system may further include regulatory elements that enhance transcriptional activity, such as minimal promoters. The inventive concept also encompasses methods for optimizing gene expression, including identifying and incorporating optimal nucleic acid sequences, and kits for expressing genes in rod photoreceptors.
Novelty and Inventive Step
The new inventive concept introduces a higher identity threshold (97% vs. 95%) for the nucleic acid sequence, ensuring more specific and efficient gene expression. Additionally, the incorporation of regulatory elements and optimization methods represents a non-obvious improvement over the original patent.
Alternative Embodiments and Variations
Alternative embodiments may include the use of different regulatory elements, such as enhancers or silencers, to modulate gene expression. Variations may also include the development of expression cassettes targeting other photoreceptor cell types or using different vector systems.
Potential Commercial Applications and Market
The enhanced gene expression system has potential applications in gene therapy, optogenetics, and regenerative medicine, particularly in the treatment of retinal diseases. The target market includes biotech and pharmaceutical companies, as well as research institutions focused on vision science and gene therapy.
CPC Classifications
| Section | Class | Group |
|---|---|---|
| A | A61 | A61K48/0058 |
| A | A61 | A61K48/00 |
| C | C12 | C12N15/63 |
| C | C12 | C12N15/79 |
| C | C12 | C12N15/86 |
| C | C12 | C12N2750/14143 |
| C | C12 | C12N2830/008 |
Section 103 Obviousness Analysis (PHOSITA)
Field of Art
Molecular Biology and Gene Expression, specifically focused on nucleic acid sequence design for targeted photoreceptor cell gene expression, requiring expertise in molecular genetics, sequence analysis, promoter engineering, and viral vector technologies
Person of Ordinary Skill (PHOSITA) Profile
A molecular biologist with advanced degree (PhD) and 3-5 years specialized experience in genetic engineering, familiar with sequence manipulation, promoter design, gene expression systems, and rod photoreceptor targeting techniques
Obviousness Rationale
A skilled practitioner would recognize that incrementally modifying the source patent's nucleic acid sequence identity threshold and incorporating additional regulatory elements represents a predictable optimization strategy in gene expression system design. The variations disclosed in the PTD represent routine engineering modifications that would be apparent to one skilled in molecular genetics and promoter engineering. The core technical concept of rod photoreceptor-specific gene expression remains consistent between the source patent and PTD, with the variations representing incremental improvements rather than breakthrough innovations.
Obvious Combinations & Variations
Original Patent Information
| Patent Number | US 11,857,642 |
|---|---|
| Title | SYNP161, a promoter for the expression of genes |
| Assignee(s) | FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESEARCH |