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1. WO2022099034 - MITOCHONDRIAL PROTEIN TARGETING ENGINEERED DEUBIQUITINASES AND METHODS OF USE THEREOF

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Publication Number WO/2022/099034
Publication Date 12.05.2022
International Application No. PCT/US2021/058286
International Filing Date 05.11.2021
IPC
C07K 14/415 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
415from plants
C12N 9/22 2006.1
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 15/86 2006.1
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
Applicants
  • PETRICHOR HEALTHCARE CAPITAL MANAGEMENT LP [US]/[US]
Inventors
  • LOEW, Andreas
  • HALL, Samuel W.
Agents
  • GRANT, Angela M.
Priority Data
63/110,62506.11.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) MITOCHONDRIAL PROTEIN TARGETING ENGINEERED DEUBIQUITINASES AND METHODS OF USE THEREOF
(FR) DÉSUBIQUITINASES MODIFIÉES CIBLANT UNE PROTÉINE MITOCHONDRIALE ET LEURS MÉTHODES D'UTILISATION
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Abstract
(EN) Provided herein are fusion protein comprising: an effector domain comprising a catalytic domain of a deubiquitinase, or a functional fragment or functional variant thereof; and a targeting domain comprising a moiety that specifically binds a mitochondrial protein. Also provided herein are methods of using the fusion proteins to treat a disease, including genetic diseases.
(FR) La présente invention concerne une protéine de fusion comprenant : un domaine effecteur comprenant un domaine catalytique d'une désubiquitinase, ou un fragment fonctionnel ou un variant fonctionnel associé ; et un domaine de ciblage comprenant une fraction qui se lie spécifiquement à une protéine mitochondriale. L'invention concerne également des méthodes d'utilisation des protéines de fusion pour traiter une maladie, notamment des maladies génétiques.
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