THERAPEUTIC SPLICE-SWITCHING OLIGONUCLEOTIDES

The present disclosure provides compositions and methods for treating a disorder associated with mutations in the CEP290 gene. The disclosure includes synthetic polynucleotides for skipping a reading-frame of a CEP290 pre-RNA, yielding a CEP290 translated product that lacks one or more exons. The di...

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Bibliographic Details
Main Authors WIENHOLDS, ERNO, SUN, MARK GEORGE FORD, GONCALVES, JOAO ANTONIO LOURENCO, MERICO, DANIELE
Format Patent
LanguageEnglish
French
Published 27.02.2020
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
SUGARS
VINEGAR
WINE
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Summary:The present disclosure provides compositions and methods for treating a disorder associated with mutations in the CEP290 gene. The disclosure includes synthetic polynucleotides for skipping a reading-frame of a CEP290 pre-RNA, yielding a CEP290 translated product that lacks one or more exons. The disclosure also provides methods of treating patients with the synthetic polynucleotides disclosed herein. La présente invention concerne des compositions et des méthodes pour le traitement d'un trouble associé à des mutations dans le gène CEP290. L'invention comprend des polynucléotides synthétiques pour sauter un cadre de lecture d'un pré-ARN de CEP290, produisant un produit traduit de CEP290 qui est dépourvu d'un ou de plusieurs exons. L'invention concerne également des méthodes de traitement de patients avec les polynucléotides synthétiques décrits ici.
Bibliography:Application Number: CA20193107890