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Novel Approach to Rendering Nucleic Acids Resistant to Nuclease Digestion that Maintains Sequence Selective Hybridization for Gene Therapy

详细技术说明

UC San Diego researchers are patenting a novel approach to pack nucleic acids into polymeric supramolecular nanostructures and render them resistant to nuclease digestion while maintaining sequence selective hybridization competency. This technology is predicated on the idea that steric hindrance through dense packaging limits the accessibility of DNA to selective and non-selective nucleases, while this morphology continues to allow access to additional complementary DNA strands, thus preserving the inherent sequence-selective binding and recognition properties. By packaging and delivering nucleic acids for in vivo applications while preserving the integrity of the base-sequence exposed to nuclease-rich environments, this technology will facilitate more potent and selective communication with important cellular machinery.Features: • Delivers small, modified, nucleic acids to a specific cell type. • Is non-toxic compared to other commercially available compounds.• Offers specificity, delivering the nucleic acid to only cells that are externally expressing particular receptors. • Amenable for use—biocompatible, all-in-one package (without transfection reagents) for tuning and increasing resistance to nucleases.

附加资料

Inventor: TATRO, Erick T. | GIANNESCHI, Nathan | RUSH, Anthony M.
Priority Number: WO2014047649A1
IPC Current: C12N001511 | A61K004800
Assignee Applicant: The Regents of the University of California
Title: METHODS FOR ARRANGING AND PACKING NUCLEIC ACIDS FOR UNUSUAL RESISTANCE TO NUCLEASES AND TARGETED DELIVERY FOR GENE THERAPY | PROCÉDÉS D'AGENCEMENT ET D'EMPAQUETAGE D'ACIDES NUCLÉIQUES POUR UNE RÉSISTANCE INHABITUELLE VIS-À-VIS DE NUCLÉASES ET UNE ADMINIST
Usefulness: METHODS FOR ARRANGING AND PACKING NUCLEIC ACIDS FOR UNUSUAL RESISTANCE TO NUCLEASES AND TARGETED DELIVERY FOR GENE THERAPY | PROCÉDÉS D'AGENCEMENT ET D'EMPAQUETAGE D'ACIDES NUCLÉIQUES POUR UNE RÉSISTANCE INHABITUELLE VIS-À-VIS DE NUCLÉASES ET UNE ADMINISTRATION CIBLÉE POUR UNE THÉRAPIE GÉNIQUE
Summary: The nucleic acid conjugate, targeted polymer nanoparticle, micelle, and formulation are useful for delivering oligonucleotide into a target cell (claimed) and for treating diseases and disorders including cancer, a proliferative disease or disorder; apoptosis, a disease or disorder associated with impaired apoptosis regulation, diabetes, a cardiovascular disease or disorder, a lysosomal storage disorder, an inflammatory disease or disorder (e.g., inflammatory bowel disease, including Crohn's disease, ulcerative colitis, psoriasis, arthritis etc.), inflammation; a metabolic disease or disorder, metabolic syndrome, insulin resistance syndrome, diabetes, hyperlipidemia, fatty liver disease, cachexia, obesity, atherosclerosis, arterioscerlosis, a disease disorder or condition associated with altered cholesterol metabolism, elevated blood pressure, a neurological disease or disorder; a disease or disorder associated with impaired neuronal cell differentiation; a disease or disorder associated with impaired neurite formation; a disease or disorder associated with impaired glycoprotein desialylation and an infection (e.g., viral infection, systemic infection or any other type of infection); HIV infection (e.g., in AIDS), Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis, arthritis, cystic fibrosis, hemophilia, Tay Sachs disease, and mood disorder.
Novelty: New nucleic acid conjugate comprising an oligonucleotide complementary to a target sequence associated with aberrant physiological activity and a hydrophobic polymer, useful for delivering oligonucleotide into a target cell