WO2023178311A1 - Procédés de traitement de troubles sous hypothermie et compositions associées - Google Patents

Procédés de traitement de troubles sous hypothermie et compositions associées Download PDF

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Publication number
WO2023178311A1
WO2023178311A1 PCT/US2023/064633 US2023064633W WO2023178311A1 WO 2023178311 A1 WO2023178311 A1 WO 2023178311A1 US 2023064633 W US2023064633 W US 2023064633W WO 2023178311 A1 WO2023178311 A1 WO 2023178311A1
Authority
WO
WIPO (PCT)
Prior art keywords
fold
subject
nucleic acid
cell
aav
Prior art date
Application number
PCT/US2023/064633
Other languages
English (en)
Inventor
Bin Xiao
Xiao Xiao
Juan Li
Original Assignee
The University Of North Carolina At Chapel Hill
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 The University Of North Carolina At Chapel Hill filed Critical The University Of North Carolina At Chapel Hill
Publication of WO2023178311A1 publication Critical patent/WO2023178311A1/fr

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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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61F—FILTERS IMPLANTABLE INTO BLOOD VESSELS; PROSTHESES; DEVICES PROVIDING PATENCY TO, OR PREVENTING COLLAPSING OF, TUBULAR STRUCTURES OF THE BODY, e.g. STENTS; ORTHOPAEDIC, NURSING OR CONTRACEPTIVE DEVICES; FOMENTATION; TREATMENT OR PROTECTION OF EYES OR EARS; BANDAGES, DRESSINGS OR ABSORBENT PADS; FIRST-AID KITS
    • A61F7/00—Heating or cooling appliances for medical or therapeutic treatment of the human body
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne des procédés comprenant la soumission d'une cellule ou d'un sujet à une température hypothermique. Les procédés de l'invention comprennent des procédés d'administration d'un acide nucléique dans une cellule, d'expression d'un gène synthétique ou d'un produit génique dans une cellule, d'administration d'une molécule d'acide nucléique à travers une barrière hémato-encéphalique chez un sujet, d'amélioration de l'administration d'acide nucléique à travers une barrière hémato-encéphalique chez un sujet, de traitement d'un trouble et/ou d'amélioration du traitement d'un trouble associé à une expression et/ou une activité aberrante d'un gène ou d'un produit génique chez un sujet en ayant besoin, de tels troubles du SNC comprenant, mais sans y être limités, la sclérose latérale amyotrophique.
PCT/US2023/064633 2022-03-18 2023-03-17 Procédés de traitement de troubles sous hypothermie et compositions associées WO2023178311A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263321371P 2022-03-18 2022-03-18
US63/321,371 2022-03-18

Publications (1)

Publication Number Publication Date
WO2023178311A1 true WO2023178311A1 (fr) 2023-09-21

Family

ID=88024526

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/064633 WO2023178311A1 (fr) 2022-03-18 2023-03-17 Procédés de traitement de troubles sous hypothermie et compositions associées

Country Status (1)

Country Link
WO (1) WO2023178311A1 (fr)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6632427B1 (en) * 1994-12-13 2003-10-14 Aventis Pharma S.A. Adenoviral-vector-mediated gene transfer into medullary motor neurons
WO2004049901A2 (fr) * 2002-11-27 2004-06-17 The University Of North Carolina At Chapel Hill Procedes relatifs au traitement de l'accident ischemique transitoire
WO2011117258A2 (fr) * 2010-03-22 2011-09-29 Association Institut De Myologie Procédés d'augmentation de l'efficacité de pénétration d'un vecteur dans un tissu cible
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US20200325456A1 (en) * 2017-12-19 2020-10-15 The University Of North Carolina At Chapel Hill Methods and compositions for delivery of viral vectors across the blood-brain barrier

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6632427B1 (en) * 1994-12-13 2003-10-14 Aventis Pharma S.A. Adenoviral-vector-mediated gene transfer into medullary motor neurons
WO2004049901A2 (fr) * 2002-11-27 2004-06-17 The University Of North Carolina At Chapel Hill Procedes relatifs au traitement de l'accident ischemique transitoire
WO2011117258A2 (fr) * 2010-03-22 2011-09-29 Association Institut De Myologie Procédés d'augmentation de l'efficacité de pénétration d'un vecteur dans un tissu cible
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US20200325456A1 (en) * 2017-12-19 2020-10-15 The University Of North Carolina At Chapel Hill Methods and compositions for delivery of viral vectors across the blood-brain barrier

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