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WO2016183593A3 - Prenatal therapy - Google Patents

Prenatal therapy Download PDF

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Publication number
WO2016183593A3
WO2016183593A3 PCT/US2016/032785 US2016032785W WO2016183593A3 WO 2016183593 A3 WO2016183593 A3 WO 2016183593A3 US 2016032785 W US2016032785 W US 2016032785W WO 2016183593 A3 WO2016183593 A3 WO 2016183593A3
Authority
WO
WIPO (PCT)
Prior art keywords
subject
term
stem cell
derived stem
placenta
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2016/032785
Other languages
French (fr)
Other versions
WO2016183593A2 (en
Inventor
Aijun Wang
Maricel MIGUELINO
Jerry Powell
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California Berkeley
University of California San Diego UCSD
Original Assignee
University of California Berkeley
University of California San Diego UCSD
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 University of California Berkeley, University of California San Diego UCSD filed Critical University of California Berkeley
Publication of WO2016183593A2 publication Critical patent/WO2016183593A2/en
Publication of WO2016183593A3 publication Critical patent/WO2016183593A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Compositions and methods are provided to treat or prevent a genetic disease or disorder, e.g., Hemophilia A, in a subject by administering to the subject an effective amount of a polynucleotide encoding a nucleic acid encoding a functional Factor VIII polypeptide in a pre-term placenta-derived stem cell. The subject to be treated can be a fetus and the cell is a pre-term chorionic villus tissue derived stem cell (CSC) that has been isolated from the placenta before the baby is born.
PCT/US2016/032785 2015-05-14 2016-05-16 Prenatal therapy Ceased WO2016183593A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562161793P 2015-05-14 2015-05-14
US62/161,793 2015-05-14

Publications (2)

Publication Number Publication Date
WO2016183593A2 WO2016183593A2 (en) 2016-11-17
WO2016183593A3 true WO2016183593A3 (en) 2017-03-09

Family

ID=57249407

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/032785 Ceased WO2016183593A2 (en) 2015-05-14 2016-05-16 Prenatal therapy

Country Status (1)

Country Link
WO (1) WO2016183593A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL320371A (en) * 2016-09-08 2025-06-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Gene therapy for patients with Fanconi anemia
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES
CA3073678A1 (en) * 2017-08-23 2019-02-28 Wake Forest University Health Sciences Post-natal transplantation of factor viii-expressing cells for treatment of hemophilia
EP3672605B1 (en) * 2017-08-23 2024-10-23 Wake Forest University Health Sciences In utero transplantation of factor viii-expressing cells for treatment of hemophilia
EP3784695B1 (en) 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
WO2019210270A2 (en) * 2018-04-27 2019-10-31 Seattle Children's Hospital D/B/A Seattle Children's Research Institute In vivo gene therapy using delivery of a lentiviral gene construct

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020039572A1 (en) * 1996-04-09 2002-04-04 Karen M. Downs Chimeric mammalian allantois
US20030224508A1 (en) * 1997-12-05 2003-12-04 Ill Charles R. Novel vectors and genes exhibiting increased expression
US20050276787A1 (en) * 1998-10-20 2005-12-15 Couto Linda B Adeno-associated vector compositions for expression of factor VIII
US20060034813A1 (en) * 2003-04-15 2006-02-16 Christian Herder Expression of proteins in cord blood-derived endothelial cells
US20070042462A1 (en) * 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090148425A1 (en) * 2005-10-28 2009-06-11 Tsukasa Ohmori Therapeutic method for blood coagulation disorder
WO2010042800A1 (en) * 2008-10-10 2010-04-15 Nevada Cancer Institute Methods of reprogramming somatic cells and methods of use for such cells
US20120082987A1 (en) * 2007-12-31 2012-04-05 Baxter International Inc. Transgenic Non-Human Animals Expressing Human Blood Clotting Factors and Uses Thereof
US20130236961A1 (en) * 2010-09-07 2013-09-12 Technion Research & Development Foundation Limited Novel methods and culture media for culturing pluripotent stem cells

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020039572A1 (en) * 1996-04-09 2002-04-04 Karen M. Downs Chimeric mammalian allantois
US20030224508A1 (en) * 1997-12-05 2003-12-04 Ill Charles R. Novel vectors and genes exhibiting increased expression
US20050276787A1 (en) * 1998-10-20 2005-12-15 Couto Linda B Adeno-associated vector compositions for expression of factor VIII
US20060034813A1 (en) * 2003-04-15 2006-02-16 Christian Herder Expression of proteins in cord blood-derived endothelial cells
US20070042462A1 (en) * 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090148425A1 (en) * 2005-10-28 2009-06-11 Tsukasa Ohmori Therapeutic method for blood coagulation disorder
US20120082987A1 (en) * 2007-12-31 2012-04-05 Baxter International Inc. Transgenic Non-Human Animals Expressing Human Blood Clotting Factors and Uses Thereof
WO2010042800A1 (en) * 2008-10-10 2010-04-15 Nevada Cancer Institute Methods of reprogramming somatic cells and methods of use for such cells
US20130236961A1 (en) * 2010-09-07 2013-09-12 Technion Research & Development Foundation Limited Novel methods and culture media for culturing pluripotent stem cells

Also Published As

Publication number Publication date
WO2016183593A2 (en) 2016-11-17

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