Novel Approach to Rendering Nucleic Acids Resistant to Nuclease Digestion that Maintains Sequence Selective Hybridization for Gene Therapy
- 詳細技術說明
- 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.
- *Abstract
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Nucleic acids have exceptional potential in the preparation of complex nanostructured materials for use as therapeutic agents and as powerful investigative tools. However, unmodified nucleic acids are inherently susceptible to enzymatic degradation in biological milieu, limiting their practical utility in detection and as therapeutics in real world applications. To overcome this limitation, considerable effort has been expended toward the generation of DNA analogues capable of resisting attack. Specifically, new strategies are needed for the preparation of well-defined, stable and competent nucleic acid-based materials.
- *IP Issue Date
- Jul 9, 2015
- *Principal Investigation
-
Name: Nathan Gianneschi
Department:
Name: Erick Tatro
Department:
Name: Anthony Rush
Department:
- 附加資料
- 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
- 主要類別
- 診斷/治療
- 細分類別
- 人類免疫缺陷病毒
- 申請號碼
- 20150190525
- 其他
-
Intellectual Property Info
United States patents rights (claims have been allowed) are available for commercial development
Related Materials
Tech ID/UC Case
23489/2012-207-0
Related Cases
2012-207-0
- 國家/地區
- 美國
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