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1. WO2020205664 - COMPOSITIONS AND METHODS FOR CELLULAR REPROGRAMMING TO RESCUE VISUAL FUNCTION

Publication Number WO/2020/205664
Publication Date 08.10.2020
International Application No. PCT/US2020/025544
International Filing Date 27.03.2020
IPC
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 5/00 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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants
  • YOUHEALTH BIOTECH, LIMITED
  • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US]/[US]
Inventors
  • ZHANG, Kang
  • FU, Xiang Dong
  • FU, Xin
  • LI, Gen
Agents
  • YANG, Frank (Xiaofan)
  • YVON, Brigitte
Priority Data
62/826,77229.03.2019US
62/831,09008.04.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR CELLULAR REPROGRAMMING TO RESCUE VISUAL FUNCTION
(FR) COMPOSITIONS ET MÉTHODES DE REPROGRAMMATION CELLULAIRE POUR SAUVER LA FONCTION VISUELLE
Abstract
(EN)
Disclosed herein are methods and pharmaceutical compositions for the treatment of retinitis pigmentosa, macular degeneration and other retinal conditions by interfering with expression of genes, such as photoreceptor cell-specific nuclear receptor, neural retina-specific leucine zipper protein, and polypyrimidine-tract-binding protein in cells of the eye. These methods and compositions employ nucleic acid based therapies.
(FR)
L'invention concerne des méthodes et des compositions pharmaceutiques pour le traitement de la rétinite pigmentaire, la dégénérescence maculaire et d'autres affections rétiniennes par interférence avec l'expression de gènes, tels que le récepteur nucléaire spécifique à la cellule de photorécepteur et la protéine de type à fermeture éclair à leucine spécifique à la rétine neuronale, et la protéine de liaison au tractus polypyrimidinique dans des cellules de l'œil. Ces méthodes et compositions font appel à des thérapies basées sur des acides nucléiques.
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