Targeted Gene Regulation System with Enhanced Cellular Uptake

A next-generation system for targeted gene regulation, comprising nucleic acid conjugates with microRNAs and phosphorothioate moieties, enabling enhanced cellular uptake and stability for treating diseases and disorders.

The original patent disclosed phosphorothioate-conjugated miRNAs for treating diseases, but limitations included inefficient cellular uptake and stability. The new inventive concept addresses these limitations by introducing a system with enhanced cellular uptake and stability, enabling more effective targeted gene regulation.

The system comprises nucleic acid conjugates with microRNAs designed to selectively regulate target genes involved in diseases or disorders. The phosphorothioate moiety enhances cellular uptake and stability of the conjugate, allowing for more efficient regulation. The system can be used for treating diseases, personalized medicine, and identifying target genes for regulation.

The new claims introduce the concept of a system with enhanced cellular uptake and stability, which is not present in the original patent. The use of phosphorothioate moieties to enhance cellular uptake and stability is a non-obvious improvement over the original patent.

Alternative embodiments include using different types of nucleic acid conjugates, such as siRNAs or antisense oligonucleotides, or modifying the phosphorothioate moiety to further enhance cellular uptake and stability.

The inventive concept has significant commercial potential in the treatment of diseases and disorders, personalized medicine, and gene therapy. Target industries include pharmaceutical, biotechnology, and healthcare.