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1. WO2018169983 - METHODS OF MODULATING EXPRESSION OF TARGET NUCLEIC ACID SEQUENCES IN A CELL

Publication Number WO/2018/169983
Publication Date 20.09.2018
International Application No. PCT/US2018/022220
International Filing Date 13.03.2018
IPC
C12N 9/22 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/864 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
864Parvoviral vectors
CPC
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
C07K 14/31
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
195from bacteria
305from Micrococcaceae (F)
31from Staphylococcus (G)
C07K 14/315
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
195from bacteria
315from Streptococcus (G), e.g. Enterococci
C07K 2319/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
C07K 2319/33
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
33fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
C12N 15/111
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
111General methods applicable to biologically active non-coding nucleic acids
Applicants
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US]/[US]
Inventors
  • VORA, Suhani D.
  • CHAVEZ, Alejandro
  • CHENG, Jing
  • PRUITT, Benjamin W.
Agents
  • IWANICKI, John P.
Priority Data
62/470,53813.03.2017US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS OF MODULATING EXPRESSION OF TARGET NUCLEIC ACID SEQUENCES IN A CELL
(FR) PROCÉDÉS DE MODULATION DE L'EXPRESSION DE SÉQUENCES D'ACIDES NUCLÉIQUES CIBLES DANS UNE CELLULE
Abstract
(EN)
The present disclosure provides methods and compositions of modulating expression of a target nucleic acid sequence in a cell. The method comprises introducing into the cell a nucleic acid sequence encoding a Cas9 fusion protein and a guide RNA, wherein the Cas9 fusion protein and the guide RNA are expressed and co-localize at a target site and modulate the expression of the target nucleic acid sequence.
(FR)
La présente invention concerne des procédés et des compositions de modulation de l'expression d'une séquence d'acide nucléique cible dans une cellule. Le procédé comprend l'introduction dans la cellule d'une séquence d'acide nucléique codant pour une protéine de fusion de Cas9 et un ARN guide, la protéine de fusion Cas9 et l'ARN guide étant exprimés et colocalisés au niveau d'un site cible et modulant l'expression de la séquence d'acide nucléique cible.
Also published as
Latest bibliographic data on file with the International Bureau