Szentmiklósi, József AndrásHamed, Rashidi2017-08-232017-08-232016http://hdl.handle.net/2437/243130Currently, there is no cure for muscular dystrophy. Treatment is generally aimed at controlling the onset of symptoms to maximize the quality of life. Medical therapy contains exon-skipping agents, utrophin upregulation, histone deacetylase inhibitors, corticosteroids, myostatin blockers, ataluren, and tyrosinekinase inhibitors. Exon skipping uses antisense oligonucleotides as a treatment for genetic diseases. The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in the target RNA and restore reading frame disruption. Utrophin upregulation results improved sarcolemmal stability and prevents dystrophic pathology through a significant reduction of regeneration, necrosis and fibrosis.41enAAV – adeno-associated virusesAd – adenovirusesADSCs – adipose derived stem cellsAFOs – ancle foot orthosesBMD – Becker muscular dystrophyCVS – chorion villus samplingDAPC – dystrophin-associated protein complexDMD – Duchenne muscular dystrophyEMA – European Medical AgencyEMG – electromyographyES – human embryonicFAPs – fibro-adipogenic progenitorsHDACi – histone deacetylase inhibitorIA - intraarterialIM - intamusculariPSCs – induced pluripotent stem cellsIV – intravenousKAFOs – knee ankle foot orthosesLBM – lean body massMCK – muscle creatine kinaseMDSCs – muscle derived stem cellsMMT – manual muscle testingMSCs – mesenchymal stem cellsPMO – phosphorodiamidate morpholino oligomerPS – phosphorothioaterhBGN – recombinant human diglycanROM – range of motionROS – reactive-oxygen speciesSCs – satellita cellsSP cells – side population cellsTLRs – troll-like receptorsTMV – tight muscle volumeDEENK Témalista::Orvostudomány