NUCLEIC ACID ANTISENSE OLIGOMER READTHROUGH OF NONSENSE CODONS

This invention is related to the field of genetic engineering. In particular, it is related to compositions and methods to treat genetically-based diseases and disorder or diseases that are caused by the translation of non-functional proteins from an mRNA with a nonsense (premature) stop codon. None...

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Hauptverfasser: MORENO, Dimas, KOROSTELEV, Andrei, SERAJ, Zahra, KHVOROVA, Anatasia, SUSOROV, Denis
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creator MORENO, Dimas
KOROSTELEV, Andrei
SERAJ, Zahra
KHVOROVA, Anatasia
SUSOROV, Denis
description This invention is related to the field of genetic engineering. In particular, it is related to compositions and methods to treat genetically-based diseases and disorder or diseases that are caused by the translation of non-functional proteins from an mRNA with a nonsense (premature) stop codon. Nonesense stop codon readthrough results in a full-length protein and restores protein function. For example, a combination of a suppressor transfer ribonucleic acid (stRNA) and nucleic acid antisense oligomers are contemplated that promote translation readthrough of mRNAs with nonsense stop codons. La présente invention concerne le domaine de l'ingénierie génétique. En particulier, elle concerne des compositions et des méthodes pour traiter des maladies et des troubles d'origine génétique ou des maladies provoquées par la traduction de protéines non fonctionnelles à partir d'un ARNm contenant un codon stop non-sens (prématuré). La lecture du codon stop non-sens permet d'obtenir une protéine pleine longueur et de rétablir la fonction de la protéine. Par exemple, une combinaison d'un acide ribonucléique de transfert suppresseur (ARNst) et d'oligomères antisens d'acide nucléique est envisagée pour favoriser la lecture de la traduction des ARNm ayant des codons stop non-sens.
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title NUCLEIC ACID ANTISENSE OLIGOMER READTHROUGH OF NONSENSE CODONS
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