Processing

Please wait...

Settings

Settings

Goto Application

1. WO2020112979 - THERAPEUTIC GENE EDITING FOR ELANE-ASSOCIATED DISEASE

Publication Number WO/2020/112979
Publication Date 04.06.2020
International Application No. PCT/US2019/063578
International Filing Date 27.11.2019
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 15/10 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
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/87 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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
CPC
A61K 48/00
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
Applicants
  • BAUER, Daniel E. [US]/[US]
  • RAO, Shuquan [US]/[US]
  • NEWBURGER, Peter E. [US]/[US]
  • FRAZÃO, Josias Brito [US]/[US]
  • WOLFE, Scot A. [US]/[US]
Inventors
  • BAUER, Daniel E.
  • RAO, Shuquan
  • NEWBURGER, Peter E.
  • FRAZÃO, Josias Brito
  • WOLFE, Scot A.
Agents
  • RESNICK, David S.
  • EISENSTEIN, Ronald I.
  • FITZGERALD, Mark J.
  • BENN, Susanna C.
  • BRAICH, Ravinderjit S.
  • CROSBY, David F.
  • HUFF, Shayne Y.
  • PTASHKA, Teresa A.
  • KLING, Nicole D.
Priority Data
62/773,39730.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) THERAPEUTIC GENE EDITING FOR ELANE-ASSOCIATED DISEASE
(FR) ÉDITION DE GÈNE THÉRAPEUTIQUE POUR UNE MALADIE ASSOCIÉE À ELANE
Abstract
(EN)
Provided herein are reagents and methods for targeting the ELANE gene for inhibition. Further provided herein is a method for producing a progenitor cell or a population of progenitor cells having decreased ELANE mRNA or protein expression.
(FR)
L'invention concerne des réactifs et des procédés pour cibler le gène ELANE pour l'inhibition. L'invention concerne en outre un procédé de production d'une cellule progénitrice ou d'une population de cellules progénitrices ayant une expression de protéine ou d'ARNm ELANE réduite.
Latest bibliographic data on file with the International Bureau