MicroRNA target site for cell- or tissue-specific inhibition of expression of a transgene

Simple SummaryContent extracted from patent full text and abstract with AI.

This invention describes a specially designed microRNA-206 (miR-206) target site that can be inserted into genetic constructs to control the expression of a transgene in a cell- or tissue-specific manner. By introducing specific nucleotide substitutions into the miR-206 target site, the technology allows customized regulation of gene expression, particularly in contexts where controlling which cells express a therapeutic gene is important.

Use CasesContent extracted from patent full text and abstract with AI.

• Targeted gene therapy for diseases where tissue-specific expression of the therapeutic gene is crucial (e.g., muscle disorders, cardiac diseases).
• Development of safer genetically modified organisms (GMOs) by preventing unwanted transgene expression in non-target tissues.
• Research applications for studying the role of genes in specific cell types or tissues by selective transgene expression or silencing.
• Improved delivery of gene therapies with fewer off-target effects in clinical settings.

BenefitsContent extracted from patent full text and abstract with AI.

• Enables precise control over where and when a transgene is active in the body, reducing off-target or unintended effects.
• Improves safety and efficacy of gene therapies by minimizing transgene expression in non-target tissues.
• Provides a flexible platform for designing expression cassettes for various medical and research applications.
• Facilitates development of next-generation genetic medicines with enhanced targeting and reduced side effects.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The present invention is directed to an isolated miR-206 target site, comprising or consisting of a nucleic acid sequence with a sequence identity of at least 80% compared to wild type miR-206 target site with SEQ ID No. 1, characterized in that the nucleic acid sequence comprises at least one nucleotide substitution at a position from nucleotide 2 to 8 and/or at least one nucleotide substitution at a position from nucleotide 12 to 16 of SEQ D No. 1, wherein the nucleotide positions of SEQ ID No. 1 are numbered from the 3'- to the 5'-end; as well as to an expression cassette, vector and pharmaceutical composition comprising at least one isolated miR-206 target site of the invention.