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1. WO2020072873 - RAAV VECTORS FOR THE TREATMENT OF GM1 AND GM2 GANGLIOSIDOSIS

Publication Number WO/2020/072873
Publication Date 09.04.2020
International Application No. PCT/US2019/054649
International Filing Date 04.10.2019
IPC
C12N 15/86 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
86Viral vectors
C12N 15/79 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
C07K 14/005 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
CPC
C07K 14/005
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
C12N 15/86
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
Applicants
  • UNIVERSITY OF MASSACHUSETTS [US]/[US]
Inventors
  • ESTEVES, Miguel, Sena
Agents
  • DEYOUNG Janice Kugler
Priority Data
62/741,84805.10.2018US
62/814,58706.03.2019US
62/815,99608.03.2019US
62/826,86329.03.2019US
62/840,35929.04.2019US
62/848,85816.05.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) RAAV VECTORS FOR THE TREATMENT OF GM1 AND GM2 GANGLIOSIDOSIS
(FR) VECTEURS RAAV POUR LE TRAITEMENT DES GANGLIOSIDOSES À GM1 ET GM2
Abstract
(EN)
Aspects of the disclosure relate to compositions and methods for the treatment of lysosomal storage disorders, such as GM1 gangliosidosis, Tay Sachs disease, and Sandhoff disease. In some embodiments, the compositions comprise viral vectors encoding beta- galactosidase. In some embodiments, the compositions comprise viral vectors encoding beta- hexosaminidase subunits (e.g. HEXA, HEXB, or combinations thereof).
(FR)
Des aspects de l'invention concernent des compositions et des procédés pour le traitement de troubles du stockage lysosomal, comme la gangliosidode à GM1, la maladie de Tay Sachs, et la maladie de Sandhoff. Dans certains modes de réalisation, les compositions comprennent des vecteurs viraux codant la bêta-galactosidase. Dans certains modes de réalisation, les compositions comprennent des vecteurs viraux codant des sous-unités de bêta-hexosaminidase (par exemple, HEXA, HEXB, ou des combinaisons de celles-ci).
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