AR133098A1 - MUTANT AAV WITH CENTRAL NERVOUS SYSTEM TARGETING MOTIFS AND COMPOSITIONS CONTAINING IT - Google Patents

MUTANT AAV WITH CENTRAL NERVOUS SYSTEM TARGETING MOTIFS AND COMPOSITIONS CONTAINING IT

Info

Publication number
AR133098A1
AR133098A1 ARP240101663A ARP240101663A AR133098A1 AR 133098 A1 AR133098 A1 AR 133098A1 AR P240101663 A ARP240101663 A AR P240101663A AR P240101663 A ARP240101663 A AR P240101663A AR 133098 A1 AR133098 A1 AR 133098A1
Authority
AR
Argentina
Prior art keywords
nervous system
compositions containing
central nervous
system targeting
mutant aav
Prior art date
Application number
ARP240101663A
Other languages
Spanish (es)
Inventor
Joshua Joyner Sims
Hao Xu
Jenny A Sidrane
Rosemary Meggersee
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of AR133098A1 publication Critical patent/AR133098A1/en

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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se proporcionan en la presente composiciones que incluyen péptidos dirigidos a células endoteliales cerebrales, microvasculares cerebrales y/o péptidos dirigidos a tejidos que atraviesan la barrera hematoencefálica (BBB) unidos a los mismos o insertados en una proteína dirigida de un vector recombinante que tiene al menos un péptido exógeno que comprende Xn - n-mero - Xm. Se proporcionan composiciones que proporcionan tales conjugados, péptidos diana o vectores recombinantes que tienen una cápside o proteína de envoltura modificada, así como sus usos.Provided herein are compositions including brain endothelial cell-targeting peptides, brain microvascular, and/or tissue-targeting peptides that cross the blood-brain barrier (BBB) attached thereto or inserted into a targeting protein of a recombinant vector having at least one exogenous peptide comprising Xn-n-mer-Xm. Compositions providing such conjugates, targeting peptides, or recombinant vectors having a modified capsid or envelope protein, as well as their uses, are provided.

ARP240101663A 2023-06-29 2024-06-28 MUTANT AAV WITH CENTRAL NERVOUS SYSTEM TARGETING MOTIFS AND COMPOSITIONS CONTAINING IT AR133098A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202363511094P 2023-06-29 2023-06-29

Publications (1)

Publication Number Publication Date
AR133098A1 true AR133098A1 (en) 2025-08-27

Family

ID=91966663

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP240101663A AR133098A1 (en) 2023-06-29 2024-06-28 MUTANT AAV WITH CENTRAL NERVOUS SYSTEM TARGETING MOTIFS AND COMPOSITIONS CONTAINING IT

Country Status (3)

Country Link
AR (1) AR133098A1 (en)
TW (1) TW202516019A (en)
WO (1) WO2025007046A1 (en)

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WO2025007046A1 (en) 2025-01-02
TW202516019A (en) 2025-04-16

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