CA2948844C - Engineering synthetic brain penetrating gene vectors - Google Patents
Engineering synthetic brain penetrating gene vectors Download PDFInfo
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- CA2948844C CA2948844C CA2948844A CA2948844A CA2948844C CA 2948844 C CA2948844 C CA 2948844C CA 2948844 A CA2948844 A CA 2948844A CA 2948844 A CA2948844 A CA 2948844A CA 2948844 C CA2948844 C CA 2948844C
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- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
- A61K47/6931—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
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- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
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- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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- Biochemistry (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (7)
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| US201462001994P | 2014-05-22 | 2014-05-22 | |
| US62/001,994 | 2014-05-22 | ||
| PCT/US2015/030381 WO2015175539A1 (en) | 2014-05-12 | 2015-05-12 | Engineering synthetic brain penetrating gene vectors |
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| WO2011106702A2 (en) | 2010-02-25 | 2011-09-01 | The Johns Hopkins University | Sustained delivery of therapeutic agents to an eye compartment |
| WO2017044759A1 (en) * | 2015-09-11 | 2017-03-16 | The Johns Hopkins University | Compositions and methods to improve nanoparticle distribution within the brain interstitium |
| WO2020210805A1 (en) * | 2019-04-11 | 2020-10-15 | The Johns Hopkins University | Nanoparticles for drug delivery to brain |
| CN111658785B (zh) * | 2020-04-21 | 2023-05-12 | 威爱医疗科技(中山)有限公司 | 一种基因载体及其制备方法和应用 |
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| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
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| US5527675A (en) | 1993-08-20 | 1996-06-18 | Millipore Corporation | Method for degradation and sequencing of polymers which sequentially eliminate terminal residues |
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| US6509323B1 (en) | 1998-07-01 | 2003-01-21 | California Institute Of Technology | Linear cyclodextrin copolymers |
| CA2406081C (en) | 2000-04-26 | 2009-12-22 | Oregon Health Sciences University | Administration of a thiol-based chemoprotectant compound |
| AU2001271345A1 (en) | 2000-07-10 | 2002-01-21 | Albert T. Naito | Method for opening the blood-brain barrier |
| RU2322500C2 (ru) | 2000-12-01 | 2008-04-20 | Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. | Малые молекулы рнк, опосредующие интерференцию рнк |
| EP1648914A4 (en) | 2003-07-31 | 2009-12-16 | Regulus Therapeutics Inc | OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA |
| US8969314B2 (en) | 2003-07-31 | 2015-03-03 | Regulus Therapeutics, Inc. | Methods for use in modulating miR-122a |
| US20050142581A1 (en) | 2003-09-04 | 2005-06-30 | Griffey Richard H. | Microrna as ligands and target molecules |
| US20080213891A1 (en) | 2004-07-21 | 2008-09-04 | Alnylam Pharmaceuticals, Inc. | RNAi Agents Comprising Universal Nucleobases |
| US7579451B2 (en) | 2004-07-21 | 2009-08-25 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a modified or non-natural nucleobase |
| WO2006112872A2 (en) | 2004-08-04 | 2006-10-26 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase |
| US20060223777A1 (en) | 2005-03-29 | 2006-10-05 | Dharmacon, Inc. | Highly functional short hairpin RNA |
| US20070213292A1 (en) | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| AU2006279906B2 (en) | 2005-08-10 | 2012-05-10 | Alnylam Pharmaceuticals, Inc. | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| WO2007027894A2 (en) | 2005-08-29 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Antisense compounds having enhanced anti-microrna activity |
| US8658608B2 (en) | 2005-11-23 | 2014-02-25 | Yale University | Modified triple-helix forming oligonucleotides for targeted mutagenesis |
| EP2388327A1 (en) | 2006-01-27 | 2011-11-23 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and compositions for the use in modulation of micrornas |
| KR101407707B1 (ko) | 2006-04-03 | 2014-06-19 | 산타리스 팔마 에이/에스 | Anti-mirna 안티센스 올리고뉴클레오타이드를 함유하는 약학적 조성물 |
| JP2008053276A (ja) | 2006-08-22 | 2008-03-06 | Nec Electronics Corp | 半導体装置の製造方法 |
| EP2090665A2 (en) | 2006-10-20 | 2009-08-19 | Exiqon A/S | Novel human microRNAs associated with cancer |
| US20090137504A1 (en) | 2006-12-21 | 2009-05-28 | Soren Morgenthaler Echwald | Microrna target site blocking oligos and uses thereof |
| US20110262406A1 (en) | 2010-04-21 | 2011-10-27 | Yale University | Compositions and methods for targeted inactivation of hiv cell surface receptors |
| WO2012039979A2 (en) | 2010-09-10 | 2012-03-29 | The Johns Hopkins University | Rapid diffusion of large polymeric nanoparticles in the mammalian brain |
| CA2819635A1 (en) * | 2010-12-01 | 2012-06-07 | Spinal Modulation, Inc. | Directed delivery of agents to neural anatomy |
| WO2012109363A2 (en) * | 2011-02-08 | 2012-08-16 | The Johns Hopkins University | Mucus penetrating gene carriers |
| CN107982548A (zh) * | 2012-02-07 | 2018-05-04 | 全球生物疗法有限公司 | 核酸输送的区室化方法及其组合物和应用 |
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