TREATMENT OF GENETIC DISEASES CHARACTERIZED BY UNSTABLE mRNAs

Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceu...

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Hauptverfasser: KARNI, Rotem, AMAR-SCHWARTZ, Adi
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creator KARNI, Rotem
AMAR-SCHWARTZ, Adi
description Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both. L'invention concerne des procédés de traitement d'une maladie caractérisée par une instabilité d'ARNm ou une dégradation médiée par un non-sens d'un ARNm d'un gène associé à une maladie chez un sujet par l'administration d'une composition pharmaceutique comprenant au moins un agent qui diminue l'expression de FTO, sa fonction ou les deux. L'invention concerne également des kits et des compositions pharmaceutiques comprenant un agent qui diminue l'expression de FTO, sa fonction ou les deux et un agent favorisant la transcription ininterrompue, ainsi que des procédés de détermination de l'adéquation d'un sujet à traiter avec un agent qui diminue l'expression de FTO, sa fonction ou les deux.
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subjects HUMAN NECESSITIES
HYGIENE
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES
MEASURING
MEDICAL OR VETERINARY SCIENCE
PHYSICS
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
TESTING
title TREATMENT OF GENETIC DISEASES CHARACTERIZED BY UNSTABLE mRNAs
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