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1. WO2022126189 - ADENO-ASSOCIATED VIRUS CAPSIDS AND VECTORS

Publication Number WO/2022/126189
Publication Date 23.06.2022
International Application No. PCT/AU2021/051497
International Filing Date 16.12.2021
IPC
C07K 14/005 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
C12N 15/86 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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12N 7/00 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
7Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
Applicants
  • CHILDREN'S MEDICAL RESEARCH INSTITUTE [AU]/[AU]
Inventors
  • WESTHAUS, Adrian
  • LISOWSKI, Leszek
  • CABANES CREUS, Marti
Agents
  • DAVIES COLLISON CAVE PTY LTD
Priority Data
202090468716.12.2020AU
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) ADENO-ASSOCIATED VIRUS CAPSIDS AND VECTORS
(FR) CAPSIDES ET VECTEURS DE VIRUS ADÉNO-ASSOCIÉS
Abstract
(EN) The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells. The disclosure includes AAV capsid polypeptides comprising a peptide modification in variable region 8 (VR VIII), where the peptide modification comprises a 7 amino acid insertion.
(FR) La présente divulgation concerne d'une manière générale des polypeptides capsidiques de virus adéno-associés (VAA) et des molécules d'acide nucléique codantes. La divulgation concerne également des vecteurs de VAA comprenant les polypeptides capsidiques, et des vecteurs d'acide nucléique (par exemple, des plasmides) comprenant les molécules d'acides nucléiques codantes, ainsi que des cellules hôtes comprenant les vecteurs. La divulgation concerne en outre des procédés et des utilisations des polypeptides, des molécules d'acides nucléiques codantes, des vecteurs et des cellules hôtes. La divulgation concerne des polypeptides capsidiques de VAA comprenant une modification peptidique dans la région variable 8 (VR VIII), la modification peptidique comprenant une insertion de 7 acides aminés.
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