WO2018129203A3 - Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination - Google Patents

Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination Download PDF

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Publication number
WO2018129203A3
WO2018129203A3 PCT/US2018/012409 US2018012409W WO2018129203A3 WO 2018129203 A3 WO2018129203 A3 WO 2018129203A3 US 2018012409 W US2018012409 W US 2018012409W WO 2018129203 A3 WO2018129203 A3 WO 2018129203A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
tissue
temporal
drug delivery
specific drug
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Application number
PCT/US2018/012409
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French (fr)
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WO2018129203A2 (en
Inventor
Cheng-Han Chen
Benjamin M. Wu
Reza Ardehali
Ngoc Bao Nguyen
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The Regents Of The University Of California
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Publication of WO2018129203A2 publication Critical patent/WO2018129203A2/en
Publication of WO2018129203A3 publication Critical patent/WO2018129203A3/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • A61K47/50—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
    • A61K47/69—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
    • A61K47/6921—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
    • A61K47/6927—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
    • 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
    • A61K47/6935—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 the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol
    • A61K47/6937—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 the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol the polymer being PLGA, PLA or polyglycolic acid
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/203—Animal model comprising inducible/conditional expression system, e.g. hormones, tet
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Epidemiology (AREA)
  • Nanotechnology (AREA)
  • Animal Husbandry (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

In certain embodiments METHODS for conditional and target-specific recombination are provided where the methods comprise: providing a mammal comprising cells that express an ER-ligand-inducible CRE recombinase under the control of a promoter, and that comprises a nucleic acid sequence flanked by a pair of loxP sequences; and administering to the mammal biodegradable polymer nanoparticles containing an estrogen receptor ligand where the nanoparticles provide specific delivery to a target tissue of the estrogen receptor ligand thereby activating said CRE recombinase which performs target specific recombination of the nucleic acid between the loxP sequences in said target tissue.
PCT/US2018/012409 2017-01-06 2018-01-04 Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination WO2018129203A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762443604P 2017-01-06 2017-01-06
US62/443,604 2017-01-06

Publications (2)

Publication Number Publication Date
WO2018129203A2 WO2018129203A2 (en) 2018-07-12
WO2018129203A3 true WO2018129203A3 (en) 2018-08-16

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Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220041682A1 (en) * 2018-11-13 2022-02-10 H. Lee Moffitt Cancer Center And Research Institute, Inc. Tlr9-targeted therapeutics
WO2020247815A1 (en) * 2019-06-06 2020-12-10 Cornell University Transgenic rodent model for lung fibrosis and uses thereof
CN116064666A (en) * 2019-10-08 2023-05-05 黄菁 Method for integrating target gene fragment

Citations (10)

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WO2002028175A2 (en) * 2000-10-03 2002-04-11 Association Pour Le Developpement De La Recherche En Genetique Moleculaire (Aderegem) Transgenic mouse for targeted recombination mediated by modified cre-er
US20040023203A1 (en) * 2002-05-31 2004-02-05 Gero Miesenbock Heterologous stimulus-gated ion channels and methods of using same
US20050013869A1 (en) * 2003-07-18 2005-01-20 Chaw Cheng Shu Sustained release formulation for carbamates and a method therefor
US20070071790A1 (en) * 2005-09-28 2007-03-29 Northwestern University Biodegradable nanocomposites with enhance mechanical properties for soft tissue
WO2008112226A2 (en) * 2007-03-13 2008-09-18 Massachusetts Institute Of Technology Cre-lox based gene knockdown constructs and methods of use thereof
US20120315322A1 (en) * 2011-06-09 2012-12-13 Nnanoaxis, Llc Nanoparticle mediated gene therapy and therapeutic products for alzheimers
US20150093365A1 (en) * 2012-04-18 2015-04-02 Dana-Farber Cancer Institute, Inc. Photoactivatable caged tamoxifen and tamoxifen derivative molecules and methods of use thereof
WO2015185691A1 (en) * 2014-06-05 2015-12-10 Eth Zurich Low-leakage cellular biosensor system
US20160022835A1 (en) * 2013-03-15 2016-01-28 The Brigham And Women's Hospital, Inc. Targeted Polymeric Inflammation-Resolving Nanoparticles
US20160175462A1 (en) * 2013-06-17 2016-06-23 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002028175A2 (en) * 2000-10-03 2002-04-11 Association Pour Le Developpement De La Recherche En Genetique Moleculaire (Aderegem) Transgenic mouse for targeted recombination mediated by modified cre-er
US20040023203A1 (en) * 2002-05-31 2004-02-05 Gero Miesenbock Heterologous stimulus-gated ion channels and methods of using same
US20050013869A1 (en) * 2003-07-18 2005-01-20 Chaw Cheng Shu Sustained release formulation for carbamates and a method therefor
US20070071790A1 (en) * 2005-09-28 2007-03-29 Northwestern University Biodegradable nanocomposites with enhance mechanical properties for soft tissue
WO2008112226A2 (en) * 2007-03-13 2008-09-18 Massachusetts Institute Of Technology Cre-lox based gene knockdown constructs and methods of use thereof
US20120315322A1 (en) * 2011-06-09 2012-12-13 Nnanoaxis, Llc Nanoparticle mediated gene therapy and therapeutic products for alzheimers
US20150093365A1 (en) * 2012-04-18 2015-04-02 Dana-Farber Cancer Institute, Inc. Photoactivatable caged tamoxifen and tamoxifen derivative molecules and methods of use thereof
US20160022835A1 (en) * 2013-03-15 2016-01-28 The Brigham And Women's Hospital, Inc. Targeted Polymeric Inflammation-Resolving Nanoparticles
US20160175462A1 (en) * 2013-06-17 2016-06-23 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
WO2015185691A1 (en) * 2014-06-05 2015-12-10 Eth Zurich Low-leakage cellular biosensor system

Non-Patent Citations (3)

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Title
BLUM ET AL.: "Prevention of K-Ras- and Pten-mediated intravaginal tumors by treatment with camptothecin-loaded PLGA nanoparticles", DRUG DELIVERY AND TRANSLATIONAL RESEARCH, vol. 1, no. 5, 10 September 2011 (2011-09-10), pages 383 - 394, XP055534589 *
ICHISE ET AL.: "Establishment of a tamoxifen-inducible Cre-driver mouse strain for widespread and temporal genetic modification in adult mice", EXPERIMENTAL ANIMALS, vol. 65, no. 3, 29 July 2016 (2016-07-29), pages 231 - 244, XP055534554 *
LOPES ET AL.: "Poly(lactic acid) production for tissue engineering applications", PROCEDIA ENGINEERING, vol. 42, 25 August 2012 (2012-08-25), pages 1402 - 1413, XP055534594 *

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