[EN] TREATMENT OF GENETIC DISEASES CHARACTERIZED BY UNSTABLE mRNAs<br/>[FR] TRAITEMENT DE MALADIES GÉNÉTIQUES CARACTÉRISÉES PAR DES ARNM INSTABLES
申请人:YISSUM RES DEV CO OF HEBREW UNIV JERUSALEM LTD
公开号:WO2021059270A2
公开(公告)日:2021-04-01
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.