WO2011087804A3 - Peptide- polynucleotide compositions for transfecting a cell with dna and for treating a neurodegenerative disease - Google Patents

Peptide- polynucleotide compositions for transfecting a cell with dna and for treating a neurodegenerative disease Download PDF

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Publication number
WO2011087804A3
WO2011087804A3 PCT/US2010/061660 US2010061660W WO2011087804A3 WO 2011087804 A3 WO2011087804 A3 WO 2011087804A3 US 2010061660 W US2010061660 W US 2010061660W WO 2011087804 A3 WO2011087804 A3 WO 2011087804A3
Authority
WO
WIPO (PCT)
Prior art keywords
peptide
dna
transfecting
cell
polynucleotide compositions
Prior art date
Application number
PCT/US2010/061660
Other languages
French (fr)
Other versions
WO2011087804A2 (en
Inventor
William F Kaemmerer
Julie Ann Alkatout
Janelle L. Blum
Jennifer M. Heisel
Matthew Jolly
Michael David Kaytor
Marcy R. Weatherspoon
Original Assignee
Medtronic, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Medtronic, Inc. filed Critical Medtronic, Inc.
Publication of WO2011087804A2 publication Critical patent/WO2011087804A2/en
Publication of WO2011087804A3 publication Critical patent/WO2011087804A3/en

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Classifications

    • 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
    • A61K48/0041—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 the non-active part being polymeric
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Peptide-polynucleotide compositions and conjugates for the treatment of neurodegenerative disease are provided. The compositions and conjugates contain a peptide having one or more of a rabies virus glycoprotein domain, a nuclear localization signal domain, and a DNA condensing domain that is linked either covalently or electrostatically to polynucleotides such as shRNA. Methods of transfecting a cell with DNA, methods of increasing shRNA expression in target cells of a mammal, and methods of treatment are also provided, which include delivery of the peptide-polynucleotide compositions and conjugates of the invention.
PCT/US2010/061660 2009-12-21 2010-12-21 Peptide-polynucleotide compositions, and methods for transfecting a cell with dna and treatment of neurodegenerative disease WO2011087804A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US28879309P 2009-12-21 2009-12-21
US61/288,793 2009-12-21

Publications (2)

Publication Number Publication Date
WO2011087804A2 WO2011087804A2 (en) 2011-07-21
WO2011087804A3 true WO2011087804A3 (en) 2011-11-17

Family

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Family Applications (1)

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PCT/US2010/061660 WO2011087804A2 (en) 2009-12-21 2010-12-21 Peptide-polynucleotide compositions, and methods for transfecting a cell with dna and treatment of neurodegenerative disease

Country Status (1)

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WO (1) WO2011087804A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013075244A1 (en) 2011-11-24 2013-05-30 Pu Chen Peptide sequence design and use thereof for peptide-mediated sirna delivery
JP6386461B2 (en) 2012-10-26 2018-09-05 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． Compositions and methods for the treatment of Parkinson's disease by selective delivery of oligonucleotide molecules to specific neuronal species
EP3967705A1 (en) 2015-02-04 2022-03-16 United Arab Emirates University Rvg derived peptides
JP6661797B2 (en) 2016-11-22 2020-03-11 株式会社東芝 Nucleic acid condensation peptide, nucleic acid condensation peptide set, nucleic acid delivery carrier, nucleic acid delivery method, cell preparation method, cell detection method and kit
EP3480212B1 (en) 2017-11-03 2023-09-27 Consejo Superior De Investigaciones Científicas Il13ralpha2 peptide and its uses
WO2020008083A1 (en) 2018-07-05 2020-01-09 Consejo Superior De Investigaciones Científicas Therapeutic target in chemokine receptors for the screening of compounds useful for the treatment of pathological processes involving chemokine signaling

Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2008054544A2 (en) * 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier

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US109139A (en) 1870-11-08 Improvement in wood pavements
US4350159A (en) 1980-02-29 1982-09-21 Gouda Kasim I Frame for stereotactic surgery
US4692147A (en) 1980-04-02 1987-09-08 Medtronic, Inc. Drug administration device
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US20050048641A1 (en) 2002-11-26 2005-03-03 Medtronic, Inc. System and method for delivering polynucleotides to the central nervous system
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US20050065760A1 (en) 2003-09-23 2005-03-24 Robert Murtfeldt Method for advising patients concerning doses of insulin

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008054544A2 (en) * 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
GRAMLICH PHILIPP M E ET AL: "Postsynthetic DNA modification through the copper-catalyzed azide-alkyne cycloaddition reaction", ANGEWANDTE CHEMIE. INTERNATIONAL EDITION, WILEY VCH VERLAG, WEINHEIM, vol. 47, no. 44, 20 October 2008 (2008-10-20), pages 8350 - 8358, XP009120328, ISSN: 1433-7851, [retrieved on 20080922], DOI: DOI:10.1002/ANIE.200802077 *
KUMAR PRITI ET AL: "Transvascular delivery of small interfering RNA to the central nervous system", NATURE, NATURE PUBLISHING GROUP, LONDON, GB, vol. 448, no. 7149, 5 July 2007 (2007-07-05), pages 39 - 45, XP002499820, ISSN: 0028-0836, DOI: DOI:10.1038/NATURE05901 *
LIU Y ET AL: "Brain-targeting gene delivery and cellular internalization mechanisms for modified rabies virus glycoprotein RVG29 nanoparticles", BIOMATERIALS, ELSEVIER SCIENCE PUBLISHERS BV., BARKING, GB, vol. 30, no. 25, 1 September 2009 (2009-09-01), pages 4195 - 4202, XP026338546, ISSN: 0142-9612, [retrieved on 20090520], DOI: DOI:10.1016/J.BIOMATERIALS.2009.02.051 *

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