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1. WO2021011840 - COMPOSITIONS AND PRODUCTION OF NICKED CLOSED-ENDED DNA VECTORS

Publication Number WO/2021/011840
Publication Date 21.01.2021
International Application No. PCT/US2020/042445
International Filing Date 17.07.2020
IPC
C12N 15/63 2006.01
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 15/09 2006.01
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
C12N 15/85 2006.01
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
Applicants
  • GENERATION BIO CO. [US]/[US]
Inventors
  • ALKAN, Ozan
  • KOTIN, Robert, Michael
  • KERR, Douglas, Anthony
  • MONDS, Russell
  • PELLETIER, Carolyn
  • STANTON, Matthew
Agents
  • CLARKE, Marcie, B.
  • BLAZESKI, George
  • BAI, Mei
  • BOWLES, Anita, M.
  • BENNETT, Coalton
Priority Data
62/875,26217.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND PRODUCTION OF NICKED CLOSED-ENDED DNA VECTORS
(FR) COMPOSITIONS ET PRODUCTION DE VECTEURS D'ADN À EXTRÉMITÉS FERMÉES NICKELÉS
Abstract
(EN)
The present application discloses methods for synthetic production and cell-free synthesis of DNA vectors, particularly closed-ended linear DNA vectors having one or more gaps (e.g., nicked ceDNA vectors, "neDNA") and adenoassociated-virus (AAV) vector which is single strand DNA having linear and continuous structure, for delivery and expression of a transgene in the host cell. The present invention also relates to an in vitro process for production of closed-ended DNA vectors, corresponding DNA vector products produced by the methods and uses thereof, and oligonucleotides and kits useful in the process of the present invention.
(FR)
La présente invention concerne des procédés de production synthétique et de synthèse acellulaire de vecteurs d'ADN, en particulier de vecteurs d'ADN linéaires à extrémités fermées ayant un ou plusieurs espaces (par exemple, des vecteurs d'ADN à extrémités fermées nickelés, « neDNA ») et un vecteur de virus adéno-associé (AAV) qui est un ADN simple brin ayant une structure linéaire et continue, pour l'administration et l'expression d'un transgène dans la cellule hôte. La présente invention concerne un processus in vitro destiné à la production de vecteurs d'ADN à extrémités fermées, des produits de vecteurs d'ADN correspondants produits par les procédés et leurs utilisations, et des oligonucléotides et des kits utiles dans le processus de l'invention.
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