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Modulacija povezovanja eksonov s protismiselnimi oligonukleotidi kot terapevtska strategija = Splicing modulation with antisense oligonucleotides as a therapeutic strategyBratkovič, Tomaž, farmacevt, 1978-Številne genske bolezni so posledica mutacij, ki vodijo v napake pri procesiranju primarnih genskih prepisov (pre-mRNA). Izrezovanje intronov in povezovanje eksonov katalizira ribonukleoproteinski ... kompleks, imenovan spajalno telesce, proces pa nadzirajo številni proteinski dejavniki, ki se vežejo na regulatorna zaporedja pre-mRNA in bodisi spodbujajo ali zavirajo vključevanje posameznih eksonov v zrelo mRNA. S sinteznimi oligonukleotidi, komplementarnimi regulatornim zaporedjem na premRNA, lahko zelo specifično zaviramo vezavo proteinskih dejavnikov in posledično vplivamo na profil povezovanja eksonov (ter s tem na strukturo in funkcijo proteinskega produkta) s ciljem doseči terapevtski izid. Nedavno sta Evropska agencija za zdravila (EMA) in ameriška Agencija za hrano in zdravila (FDA) odobrili uporabo prvih terapevtskih protismiselnih oligonukleotidov, ki modulirata proces povezovanja eksonov, za zdravljenje spinalne mišične atrofije in Duchennove mišične distrofije. V prispevku je podrobneje predstavljen terapevtski potencial te nove skupine učinkovin, njihovi mehanizmi delovanj ter omejitve, s katerimi se srečujejo pri razvoju.Source: Farmacevtski vestnik : strokovno glasilo slovenske farmacije = pharmaceutical journal of Slovenia. - ISSN 0014-8229 (Letn. 70, št. 1, apr. 2019, str. 57-68)Type of material - article, component partPublish date - 2019Language - slovenianCOBISS.SI-ID - 4708209
source: Farmacevtski vestnik : strokovno glasilo slovenske farmacije = pharmaceutical journal of Slovenia. - ISSN 0014-8229 (Letn. 70, št. 1, apr. 2019, str. 57-68)
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Bratkovič, Tomaž, farmacevt, 1978- | 23598 |
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