Small interfering rna (sirna) for the therapy of type 2 (ado2) autosomal dominant osteopetrosis caused by clcn7 (ado2 clcn7-dependent) gene mutation

Petit arn interferent (arnsi) utilisable en vue du traitement de l'osteoporose autosomique dominante de type 2 (ado2) provoquee par une mutation du gene clcn7 (ado2 dependant de clcn7)


The present invention lies in the field of molecules known as "small interfering RNA" with therapeutic applications. siRNAs have the ability to reduce gene expression in an extremely specific way (1). These are small sequences of double-strand RNA, normally used in laboratory to modify cell function, which revolutionized cell biology by allowing previously precluded molecular manipulations.




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