Enhanced Rod Photoreceptor-Specific Gene Expression System

Publication ID: 24-11857642_0006_PTD

Published: October 28, 2025

Category:Direct Improvements & Enhancements

Legal Citation

pr1or.art Inc., “Enhanced Rod Photoreceptor-Specific Gene Expression System,” Published Technical Disclosure No. 24-11857642_0006_PTD, Published October 28, 2025, available at https://archive.pr1or.art/24-11857642_0006_PTD

This technical disclosure describes improvements that would be readily apparent to a Person Having Ordinary Skill In The Art (PHOSITA) when considered in combination with the foundational architecture disclosed in U.S. Patent No. 11,857,642.

Summary of the Inventive Concept

An improved system for targeted gene expression in rod photoreceptors, featuring a novel combination of nucleic acid sequences and gene editing tools to enhance efficiency and specificity.

Background and Problem Solved

The original patent disclosed a promoter sequence for gene expression in rod photoreceptors, but it had limitations in terms of efficiency and specificity. The new inventive concept addresses these limitations by introducing a system that combines the promoter sequence with gene editing tools, such as CRISPR-Cas9 or TALEN, to enhance targeted gene expression.

Detailed Description of the Inventive Concept

The new system comprises an isolated nucleic acid molecule with a nucleic acid sequence of at least 150 base pairs and having at least 95% identity to the sequence of SEQ ID NO:1, which is specifically expressed in rod photoreceptors. The system further includes a nucleic acid sequence encoding for a gene, and optionally, a gene editing tool. The gene editing tool can be CRISPR-Cas9 or TALEN, which enables precise editing of the target gene. The system can be delivered using a combination of viral and non-viral vectors to enhance delivery efficiency. The method of using this system involves administering the expression cassette to a subject, where it is specifically expressed in rod photoreceptors and treats rod photoreceptor-related disorders.

Novelty and Inventive Step

The new claims introduce the combination of the promoter sequence with gene editing tools, which is not obvious from the original patent. This combination provides a significant improvement in targeted gene expression, making it a novel and non-obvious invention.

Alternative Embodiments and Variations

Alternative embodiments of the inventive concept could include using different gene editing tools, such as zinc finger nucleases or homology-directed repair, or using different vector systems, such as lipofection or electroporation. Variations could also include using the system for gene therapy or gene editing in other cell types.

Potential Commercial Applications and Market

The enhanced rod photoreceptor-specific gene expression system has significant commercial potential in the gene therapy and gene editing markets, particularly for treating rod photoreceptor-related disorders, such as retinitis pigmentosa or age-related macular degeneration. The market for gene therapy and gene editing is rapidly growing, and this inventive concept could provide a competitive advantage in this space.

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)

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Field of Art

Molecular Biology and Gene Expression, specifically focused on promoter sequences, gene therapy, and targeted cellular expression systems for photoreceptor cells

Person of Ordinary Skill (PHOSITA) Profile

A molecular biologist with expertise in genetic engineering, gene expression techniques, viral vector design, and cell-specific targeting strategies, holding a PhD or equivalent advanced degree with 3-5 years of specialized research experience

Obviousness Rationale

A person of ordinary skill would recognize that combining the source patent's rod photoreceptor-specific promoter sequence with emerging gene editing technologies represents a predictable extension of existing genetic engineering techniques. The PTD's integration of CRISPR-Cas9 and TALEN with the promoter sequence follows established methodological approaches in targeted gene expression. The variations demonstrate standard engineering problem-solving by applying known gene editing tools to a specific cellular targeting system.

Obvious Combinations & Variations

Source Patent Element

Isolated nucleic acid molecule with rod photoreceptor-specific sequence

PTD Variation

Adding gene editing tools like CRISPR-Cas9 and TALEN to the expression system

Obviousness Reasoning

Gene editing tools were well-known in the art for precise genetic manipulation, making their combination with cell-specific promoters a predictable design choice with expected results

Source Patent Element

Viral vector delivery mechanism

PTD Variation

Combining viral and non-viral vector delivery strategies

Obviousness Reasoning

Multi-modal delivery approaches were standard practice for enhancing gene transfer efficiency, representing an obvious optimization technique

Source Patent Element

Basic expression cassette for gene expression

PTD Variation

Expanding the system to include therapeutic applications for rod photoreceptor disorders

Obviousness Reasoning

Translating genetic research into therapeutic contexts is a standard progression in molecular biology, particularly for inherited genetic conditions

Source Patent Element

Sequence with 95% identity to SEQ ID NO:1

PTD Variation

Introducing alternative gene editing mechanisms like zinc finger nucleases

Obviousness Reasoning

Substituting equivalent gene editing technologies represents a routine engineering variation with predictable outcomes

Source Patent Element

Rod photoreceptor-specific gene expression

PTD Variation

Creating a comprehensive kit with detailed transfection protocols

Obviousness Reasoning

Developing standardized research kits with comprehensive instructions is a common practice for facilitating scientific reproducibility

35 U.S.C. § 103 Summary: Pursuant to 35 U.S.C. § 103, the variations disclosed in the instant publication would have been obvious to a person having ordinary skill in the art at the time of the invention, with a reasonable expectation of success, when considering the teachings of US Patent 11857642 in combination with contemporaneous molecular biology techniques. The disclosed system represents an obvious extension of known genetic engineering principles, combining cell-specific promoter sequences with standard gene editing and delivery methodologies.

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