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1. WO2021092518 - TRANSGENE CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR THE EXPRESSION OF HUMAN CODON-OPTIMIZED SLC6A1

Publication Number WO/2021/092518
Publication Date 14.05.2021
International Application No. PCT/US2020/059576
International Filing Date 07.11.2020
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
A61K 35/30 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
CPC
A61K 38/1787
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
177Receptors; Cell surface antigens; Cell surface determinants
1787for neuromediators, e.g. serotonin receptor, dopamine receptor
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
A61K 48/0083
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
0083characterised by an aspect of the administration regime
A61K 9/0085
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
0012Galenical forms characterised by the site of application
0085Brain, e.g. brain implants; Spinal cord
C07K 14/70571
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
70571for neuromediators, e.g. serotonin receptor, dopamine receptor
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
  • THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS [US]/[US]
Inventors
  • GRAY, Steven, J.
  • SHAFFO, Frances, C.
Agents
  • HILLMAN, Lisa, M.W.
  • GANGULY, Amritaa
  • CONRAD, Zachary
  • BIFANO, Larissa
  • GAO, Xin
Priority Data
62/932,82408.11.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) TRANSGENE CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR THE EXPRESSION OF HUMAN CODON-OPTIMIZED SLC6A1
(FR) CASSETTES TRANSGÉNIQUES, VECTEURS VAA ET VECTEURS VIRAUX VAA POUR L'EXPRESSION DU GÈNE SLC6A1 À CODONS HUMAINS OPTIMISÉS
Abstract
(EN)
The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.
(FR)
La présente invention concerne des méthodes et des compositions pour le traitement de maladies et de troubles génétiques liés à une perte et/ou une dysfonction de SLC6A1. Les méthodes et les compositions de la présente invention font appel à des vecteurs VAAr et des vecteurs viraux VAAr comprenant des molécules d'acide nucléique transgéniques comprenant des séquences d'acide nucléique codant pour un polypeptide GAT1.
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