MX367100B - Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. - Google Patents

Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.

Info

Publication number
MX367100B
MX367100B MX2014009975A MX2014009975A MX367100B MX 367100 B MX367100 B MX 367100B MX 2014009975 A MX2014009975 A MX 2014009975A MX 2014009975 A MX2014009975 A MX 2014009975A MX 367100 B MX367100 B MX 367100B
Authority
MX
Mexico
Prior art keywords
aav
organs
tissues
cells
gene transfer
Prior art date
Application number
MX2014009975A
Other languages
English (en)
Other versions
MX2014009975A (es
Inventor
A High Katherine
Mingozzi Federico
Sun Junwei
Johnson Philip
Original Assignee
The Children´S Hospital Of Philadelphia
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48984822&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX367100(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by The Children´S Hospital Of Philadelphia filed Critical The Children´S Hospital Of Philadelphia
Publication of MX2014009975A publication Critical patent/MX2014009975A/es
Publication of MX367100B publication Critical patent/MX367100B/es

Links

Classifications

    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención se refiere a un vector de virus adenoasociado (AAV), para usarse en un método de tratamiento de una enfermedad de sangrado en un mamífero, dicho vector comprendiendo una secuencia de VP1 establecida como la SEQ ID NO: 1 una secuencia de VP2 establecida como la SEQ ID NO: 3 y una secuencia de VP3 establecida como la SEQ ID NO: 4, dicho vector además comprende una secuencia polinucleótida heteróloga que comprende un gen codificando un factor de coagulación sanguínea, en donde la secuencia polinucleótida heteróloga está vinculada de forma operacional a un elemento de control de expresión que confiere la transcripción de dicha secuencia polinueclótida lheteróloga y en donde el elemento de control de expresión está activo en el hígado y en donde el método comprende administrar el vector AAV a un mamífero, para suministrar o transferir así la secuencia polinucléotida heteróloga en el mamífero.
MX2014009975A 2012-02-17 2013-02-19 Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. MX367100B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261600415P 2012-02-17 2012-02-17
PCT/US2013/026695 WO2013123503A1 (en) 2012-02-17 2013-02-19 Aav vector compositions and methods for gene transfer to cells, organs and tissues

Publications (2)

Publication Number Publication Date
MX2014009975A MX2014009975A (es) 2015-02-20
MX367100B true MX367100B (es) 2019-08-05

Family

ID=48984822

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2014009975A MX367100B (es) 2012-02-17 2013-02-19 Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.

Country Status (16)

Country Link
US (2) US20150111955A1 (es)
EP (1) EP2814958B1 (es)
JP (2) JP6373763B2 (es)
KR (1) KR102057540B1 (es)
CN (1) CN104520428B (es)
AU (1) AU2013221212B2 (es)
BR (1) BR112014020325A2 (es)
CA (1) CA2864879C (es)
CL (1) CL2014002198A1 (es)
ES (1) ES2752191T3 (es)
IL (1) IL234148B (es)
MX (1) MX367100B (es)
RU (1) RU2653444C2 (es)
SG (1) SG11201404956PA (es)
WO (1) WO2013123503A1 (es)
ZA (1) ZA201406065B (es)

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* Cited by examiner, † Cited by third party
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