Prof. Stefano Gustincich - Antisense long non-coding SINEUP RNAs: from molecular mechanism to therapeutics
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Natural SINEUPs are antisense long non-coding RNAs that enhance translation of sense mRNAs. Their activity depends on the combination of two domains: the overlapping region, or binding domain (BD), dictates SINEUP specificity, while an embedded inverted SINEB2 element acts as effector domain (ED) to UP-regulate mRNA translation. Their modular structure can be employed to artificially engineer their BDs and design synthetic SINEUPs to specifically enhance translation of virtually any target gene of interest.
By unveiling new cues on the molecular mechanism of SINEUP activity, I will present previously undescribed natural members of this functional class of non-coding RNAs.
By taking advantage of the design of synthetic SINEUPs, I will discuss representative examples of rescuing pathological phenotypes in patients’ derived cells and in mouse models of disease.
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