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1. WO2020198264 - MODIFIED CLEAVASES, USES THEREOF AND RELATED KITS

Publication Number WO/2020/198264
Publication Date 01.10.2020
International Application No. PCT/US2020/024521
International Filing Date 24.03.2020
IPC
C12N 9/48 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.)
48acting on peptide bonds, e.g. thromboplastin, leucine aminopeptidase (3.4)
C12N 9/64 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.)
48acting on peptide bonds, e.g. thromboplastin, leucine aminopeptidase (3.4)
50Proteinases
64derived from animal tissue, e.g. rennin
Applicants
  • ENCODIA, INC. [US]/[US]
Inventors
  • DESAI, Kevin
  • GUNDERSON, Kevin, L.
  • JAMES, Robert, C.
  • SHI, Lei
  • VERESPY, Stephen, III
Agents
  • CHEN, Peng
  • LEE, Robert
  • QIN, Letao
  • KAUFMAN, Marc
  • LIU, Zheng
Priority Data
62/823,92726.03.2019US
62/824,15726.03.2019US
62/931,73706.11.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MODIFIED CLEAVASES, USES THEREOF AND RELATED KITS
(FR) CLIVAGES MODIFIÉS, UTILISATIONS ET KITS CORRESPONDANTS
Abstract
(EN)
Compositions and methods are provided for the inhibition, treatment and/or prevention of degenerative myelopathy (DM) or amyotrophic lateral sclerosis (ALS). In accordance with the instant invention, nucleic acid molecules for inhibiting the expression of the superoxide dismutase 1 gene (SOD) are provided. In a particular embodiment, the nucleic acid molecules comprise an annealing domain operably linked to at least one effector domain, wherein the annealing domain hybridizes to the pre-mRNA of SOD (e.g., canine or human) and wherein the effector domain hybridizes to the U1 snRNA of U1 snRNP.
(FR)
L'invention concerne des compositions et procédés d'inhibition, de traitement et/ou de prévention de la myélopathie dégénérative (DM) ou de la sclérose latérale amyotrophique (SLA). Selon la présente invention, des molécules d'acide nucléique pour inhiber l'expression du gène de superoxyde dismutase 1 (SOD) sont fournies. Dans un mode de réalisation particulier, les molécules d'acide nucléique comprennent un domaine de recuit lié de manière fonctionnelle à au moins un domaine effecteur, le domaine de recuit s'hybridant au pré-ARNm de SOD (par exemple, canin ou humain) et le domaine effecteur s'hybridant au snARN U1 du snRNP U1 .
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