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1. WO2021092513 - CRISPR AND AAV STRATEGIES FOR X-LINKED JUVENILE RETINOSCHISIS THERAPY

Publication Number WO/2021/092513
Publication Date 14.05.2021
International Application No. PCT/US2020/059568
International Filing Date 07.11.2020
IPC
A61K 48/00 2006.01
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
CPC
A01K 2217/072
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217Genetically modified animals
07Animals genetically altered by homologous recombination
072maintaining or altering function, i.e. knock in
A01K 2227/105
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2227Animals characterised by species
10Mammal
105Murine
A01K 2267/0306
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
0306Animal model for genetic diseases
A01K 67/0278
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67Rearing or breeding animals, not otherwise provided for; New breeds of animals
027New breeds of vertebrates
0275Genetically modified vertebrates, e.g. transgenic
0278Humanized animals, e.g. knockin
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 48/0066
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
0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
Applicants
  • REGENERON PHARMACEUTICALS, INC. [US]/[US]
Inventors
  • HUNT, Charleen
  • LIU, Yang
  • GONG, Guochun
  • ROMANO, Carmelo
  • ZAMBROWICZ, Brian
Agents
  • SCHILLING, Stephen H.
  • LIEBESCHUETZ, Joe
  • SATHER, Susan K.
  • CHANG, Cindy
  • EKENA, Kirk
Priority Data
62/932,60808.11.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CRISPR AND AAV STRATEGIES FOR X-LINKED JUVENILE RETINOSCHISIS THERAPY
(FR) STRATÉGIES CRISPR-VAA POUR LA THÉRAPIE DU RÉTINOSCHISIS JUVÉNILE LIÉ À L'X
Abstract
(EN)
Nucleic acid constructs and compositions that allow insertion and/or expression of a retinoschisin coding sequence are provided. Nuclease agents targeting RS1 loci are provided. Compositions and methods of using such constructs for integration into a target genomic locus and/or expression in a cell are also provided. Methods of treating X‐linked juvenile retinoschisis using the nucleic acid constructs and compositions are also provided.
(FR)
L'invention concerne des constructions d'acide nucléique et des compositions qui permettent l'insertion et/ou l'expression d'une séquence de codage de la rétinoschisine. L'invention concerne également des agents nucléases ciblant des loci RS1 . L'invention concerne également des compositions et des procédés d'utilisation de telles constructions aux fins d'intégration dans un locus génomique cible et/ou d'expression dans une cellule. L'invention concerne également des procédés de traitement du rétinoschisis juvénile lié à l'X faisant appel auxdites constructions d'acide nucléique et compositions.
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