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1. WO2022047201 - GENE KNOCK-OUT FOR TREATMENT OF GLAUCOMA

Publication Number WO/2022/047201
Publication Date 03.03.2022
International Application No. PCT/US2021/048018
International Filing Date 27.08.2021
IPC
C12N 9/22 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/63 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
C12N 15/90 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
CPC
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 27/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
02Ophthalmic agents
06Antiglaucoma agents or miotics
C07K 14/47
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
C12N 15/907
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
902using homologous recombination
907in mammalian cells
C12N 2310/20
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2750/14143
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
Applicants
  • UNIVERSITY OF IOWA RESEARCH FOUNDATION [US]/[US]
  • ZHANG, Qihong [CN]/[US]
  • SEARBY, Charles C. [US]/[US]
  • SHEFFIELD, Val C. [US]/[US]
  • CARTER, Calvin S. [US]/[US]
  • HUANG, Sunny C. [US]/[US]
Inventors
  • ZHANG, Qihong
  • SEARBY, Charles C.
  • SHEFFIELD, Val C.
  • CARTER, Calvin S.
  • HUANG, Sunny C.
Agents
  • PERDOK, Monique M.,
  • ARORA, Suneel, Reg. No. 42,267
  • BEEKMAN, Marvin L., Reg. No. 38,377
  • BIANCHI, Timothy E., Reg. No. 39,610
  • BLACK, David W., Reg. No. 42,331
  • LANG, Allen R., Reg. No. 58,829
  • SCHEER, Bradley W., Reg. No. 47,059
Priority Data
63/071,16127.08.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) GENE KNOCK-OUT FOR TREATMENT OF GLAUCOMA
(FR) INVALIDATION GÉNIQUE POUR LE TRAITEMENT DU GLAUCOME
Abstract
(EN) A vector and methods of using the vector for, for example, to prevent, inhibit or treat glaucoma, decrease intraocular pressure or to reduce MYOC expression in a mammal, are provided.
(FR) Vecteur et méthodes d'utilisation du vecteur pour, par exemple, prévenir, inhiber ou traiter un glaucome, diminuer la pression intraoculaire ou réduire l'expression de MYOC chez un mammifère.
Latest bibliographic data on file with the International Bureau