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1. WO2020115106 - ANTISENSE OLIGONUCLEOTIDES RESCUE ABERRANT SPLICING OF ABCA4

Publication Number WO/2020/115106
Publication Date 11.06.2020
International Application No. PCT/EP2019/083618
International Filing Date 04.12.2019
IPC
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
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
CPC
A61K 31/7088
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
C12N 15/113
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; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
Applicants
  • STICHTING KATHOLIEKE UNIVERSITEIT [NL]/[NL]
Inventors
  • COLLIN, Robert Wilhelmus Johanna
  • GARANTO IGLESIAS, Alejandro
  • CREMERS, Franciscus Peter Maria
Agents
  • NEDERLANDSCH OCTROOIBUREAU
Priority Data
18210107.104.12.2018EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) ANTISENSE OLIGONUCLEOTIDES RESCUE ABERRANT SPLICING OF ABCA4
(FR) OLIGONUCLÉOTIDES ANTISENS CORRIGEANT L'ÉPISSAGE ABERRANT D'ABCA4
Abstract
(EN)
The present invention relates to the field of medicine. In particular, it relates tonovel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Stargardt disease.
(FR)
La présente invention concerne le domaine de la médecine. Plus particulièrement, l'invention concerne de nouveaux oligonucléotides antisens pouvant être utilisés pour traiter, prévenir et/ou retarder l'apparition de la maladie de Stargardt.
Latest bibliographic data on file with the International Bureau