195 KB21 KB200520252016Avsec, DamjanFlegar, Inesštevilka:6letnik:67str. 374-379ISSN:0014-8229COBISSID_HOST:30044711URN:URN:NBN:SI:doc-KTS8RJS4slSlovensko farmacevtsko društvoFarmacevtski vestnikgensko zdravljenjeHuntingtonova horeaproteini z domeno cinkovega prstaprotismiselni oligonukleotidRNA-interferencaGensko zdravljenje Huntingtonove bolezni| Gene therapy for Huntington's disease|Huntington's disease is a rare, incurable neurodegenerative disease of the central nervous system caused by the mutation in the HTT gene. Currently, there is no treatment that can cure or slow the course of Huntington's disease, so the treatment is aimed at ameliorating symptoms. Gene therapy is a promising new approach that targets the cause of Huntington's disease. Insertion of neuroprotective genes, gene silencing, and genome editing technologies are being investigated in the hope of proving effective in stopping progression of the disease. The article focuses on principles of this new approaches and presents the current state of development of gene therapy for Huntington's diseaseHuntingtonova bolezen je redka neozdravljiva nevrodegenerativna bolezen centralnega živčevja, ki jo povzroča mutacija v genu za huntingtin (HTT). Trenutno nimamo zdravila, ki bi ozdravilo ali upočasnilo potek bolezni, zato je zdravljenje večinoma simptomatsko. Gensko zdravljenje predstavlja nov pristop, ki cilja vzrok za nastanek te bolezni. Preučujejo vnos nevroprotektivnih genov ter tehnologije utišanja genov in urejanja genoma v upanju, da bodo ti pristopi upočasnili napredovanje bolezni. Članek se osredotoča na principe teh novih pristopov in podaja trenutno stanje razvoja genskega zdravljenja pri Huntingtonovi bolezniTEXTznanstveno časopisjejournalsSlovenian National E-content AggregatorNational and University Library of SloveniaSlovensko farmacevtsko društvo