Traitement en cours

Veuillez attendre...

Paramétrages

Paramétrages

Aller à Demande

1. WO2022212768 - PROMOTEURS COMPACTS POUR L'ÉDITION GÉNIQUE

Numéro de publication WO/2022/212768
Date de publication 06.10.2022
N° de la demande internationale PCT/US2022/022923
Date du dépôt international 31.03.2022
CIB
A61K 48/00 2006.1
ANÉCESSITÉS COURANTES DE LA VIE
61SCIENCES MÉDICALE OU VÉTÉRINAIRE; HYGIÈNE
KPRÉPARATIONS À USAGE MÉDICAL, DENTAIRE OU POUR LA TOILETTE
48Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiques; Thérapie génique
C12N 15/63 2006.1
CCHIMIE; MÉTALLURGIE
12BIOCHIMIE; BIÈRE; SPIRITUEUX; VIN; VINAIGRE; MICROBIOLOGIE; ENZYMOLOGIE; TECHNIQUES DE MUTATION OU DE GÉNÉTIQUE
NMICRO-ORGANISMES OU ENZYMES; COMPOSITIONS LES CONTENANT; CULTURE OU CONSERVATION DE MICRO-ORGANISMES; TECHNIQUES DE MUTATION OU DE GÉNÉTIQUE; MILIEUX DE CULTURE
15Techniques de mutation ou génie génétique; ADN ou ARN concernant le génie génétique, vecteurs, p.ex. plasmides, ou leur isolement, leur préparation ou leur purification; Utilisation d'hôtes pour ceux-ci
09Technologie d'ADN recombinant
63Introduction de matériel génétique étranger utilisant des vecteurs; Vecteurs; Utilisation d'hôtes pour ceux-ci; Régulation de l'expression
CPC
C12N 15/11
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
11DNA or RNA fragments; Modified forms thereof
C12N 15/63
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 2310/20
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 9/22
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
9Enzymes; Proenzymes; Compositions thereof
14Hydrolases (3)
16acting on ester bonds (3.1)
22Ribonucleases ; RNAses, DNAses
C40B 40/06
CCHEMISTRY; METALLURGY
40COMBINATORIAL TECHNOLOGY
BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES, IN SILICO LIBRARIES
40Libraries per se, e.g. arrays, mixtures
04Libraries containing only organic compounds
06Libraries containing nucleotides or polynucleotides, or derivatives thereof
Déposants
  • HUNTERIAN MEDICINE LLC [US]/[US]
Inventeurs
  • JASKULA-RANGA, Vinod
Mandataires
  • GUSTAFSON, Megan, A.
Données relatives à la priorité
63/168,76931.03.2021US
Langue de publication Anglais (en)
Langue de dépôt anglais (EN)
États désignés
Titre
(EN) COMPACT PROMOTERS FOR GENE EDITING
(FR) PROMOTEURS COMPACTS POUR L'ÉDITION GÉNIQUE
Abrégé
(EN) The invention relates generally to compact promoters and their use in gene editing e.g., for treating disease. The disclosure is based, in part, upon the discovery of compact, bidirectional promoters that can be used to express both a nuclease (e.g., a Cas9 nuclease) and a guide RNA (gRNA). For example, in certain embodiments disclosed herein, a compact, bidirectional promoter can comprise at least one regulatory element that directs expression of a gRNA in one direction and at least one regulatory element that directs expression of a nuclease in the other direction. Accordingly, the promoters disclosed herein use less space than prior art promoters, allowing both a nuclease and a gRNA to be packaged in a single vector (e.g., a plasmid or an AAV).
(FR) L'invention concerne de manière générale des promoteurs compacts et leur utilisation dans l'édition génique, par exemple, pour le traitement de maladies.
Dernières données bibliographiques dont dispose le Bureau international