POLYNUCLEOTIDE COMPOSITIONS FOR THE TREATMENT OF PREMATURE TERMINATION DISEASES

Polynucleotide compositions comprising (a) a complementary oligonucleotide comprising at least 10 consecutive nucleobases having at least 80% complementarity to a target gene having a premature UGA stop codon; (b) an optional linker selected from the group consisting of (i) an oligonucleotide and (i...

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Hauptverfasser: AGARWAL, Anurag, BADNER, Anna Maria, BRACKEN, Racquel Noh, POON, Yan Shuen, COFFEY, Eliot Leo
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creator AGARWAL, Anurag
BADNER, Anna Maria
BRACKEN, Racquel Noh
POON, Yan Shuen
COFFEY, Eliot Leo
description Polynucleotide compositions comprising (a) a complementary oligonucleotide comprising at least 10 consecutive nucleobases having at least 80% complementarity to a target gene having a premature UGA stop codon; (b) an optional linker selected from the group consisting of (i) an oligonucleotide and (ii) a chemical linker; and (c) an oligonucleotide comprising a SECIS element, or pharmaceutically acceptable salts thereof are disclosed herein. Les compositions polynucléotidiques comprennent (a) un oligonucléotide complémentaire comprenant au moins 10 nucléobases consécutives ayant au moins 80 % de complémentarité avec un gène cible ayant un codon d'arrêt UGA prématuré ; (b) un lieur facultatif choisi dans le groupe constitué par (i) un oligonucléotide et (ii) un lieur chimique ; et (c) un oligonucléotide comprenant un élément SECIS, ou des sels pharmaceutiquement acceptables de celui-ci.
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language eng ; fre
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS OR TEST PAPERS THEREFOR
COMPOSITIONS THEREOF
CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROCESSES OF PREPARING SUCH COMPOSITIONS
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title POLYNUCLEOTIDE COMPOSITIONS FOR THE TREATMENT OF PREMATURE TERMINATION DISEASES
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