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1. WO2020198233 - METHODS OF TREATING TNNI3-MEDIATED CARDIOMYOPATHY

Publication Number WO/2020/198233
Publication Date 01.10.2020
International Application No. PCT/US2020/024477
International Filing Date 24.03.2020
IPC
A61K 48/00 2006.01
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
C07K 14/47 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
C12N 15/86 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
Applicants
  • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US]/[US]
Inventors
  • ADLER, Eric
  • BUSHWAY, Paul
Agents
  • NOVOM, Antony
  • WAGENKNECHT, Raymond
Priority Data
62/823,46725.03.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS OF TREATING TNNI3-MEDIATED CARDIOMYOPATHY
(FR) PROCÉDÉS DE TRAITEMENT DE CARDIOMYOPATHIE MÉDIÉE PAR TNNI3
Abstract
(EN)
Provided herein are gene therapy vectors containing a polynucleotide encoding a cardiac-specific promoter operably linked to a polynucleotide encoding Troponin I3, and methods of using such vectors for preventing, mitigating, ameliorating, reducing, inhibiting, eliminating and/or reversing one or more symptoms of cardiomyopathy.
(FR)
L'invention concerne des vecteurs de thérapie génique contenant un polynucléotide codant un promoteur spécifique du cœur fonctionnellement lié à un polynucléotide codant la troponine I3, et des procédés d'utilisation de ces vecteurs pour prévenir, atténuer, améliorer, réduire, inhiber, éliminer et/ou inverser un ou plusieurs symptômes de la cardiomyopathie.
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