Pharmacological treatment of Duchenne and Becker muscular dystrophies

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dc.contributor.advisorSzentmiklósi, József András
dc.contributor.advisordeptDebreceni Egyetem::Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézethu_HU
dc.contributor.authorHamed, Rashidi
dc.contributor.departmentDE--Általános Orvostudományi Karhu_HU
dc.contributor.opponentDrimba, László
dc.contributor.opponentPórszász, Róbert
dc.contributor.opponentdeptKenézy Kórház Aneszteziológiai és Intenziv Therápiás Osztályhu_HU
dc.contributor.opponentdeptDebreceni Egyetem::Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézethu_HU
dc.date.accessioned2017-08-23T09:32:51Z
dc.date.available2017-08-23T09:32:51Z
dc.date.created2016
dc.description.abstractCurrently, 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.hu_HU
dc.description.courseáltalános orvoshu_HU
dc.description.courselangangolhu_HU
dc.description.degreeegységes, osztatlanhu_HU
dc.format.extent41hu_HU
dc.identifier.urihttp://hdl.handle.net/2437/243130
dc.language.isoenhu_HU
dc.subjectAAV – adeno-associated viruseshu_HU
dc.subjectAd – adenoviruseshu_HU
dc.subjectADSCs – adipose derived stem cellshu_HU
dc.subjectAFOs – ancle foot orthoseshu_HU
dc.subjectBMD – Becker muscular dystrophyhu_HU
dc.subjectCVS – chorion villus samplinghu_HU
dc.subjectDAPC – dystrophin-associated protein complexhu_HU
dc.subjectDMD – Duchenne muscular dystrophyhu_HU
dc.subjectEMA – European Medical Agencyhu_HU
dc.subjectEMG – electromyographyhu_HU
dc.subjectES – human embryonichu_HU
dc.subjectFAPs – fibro-adipogenic progenitorshu_HU
dc.subjectHDACi – histone deacetylase inhibitorhu_HU
dc.subjectIA - intraarterialhu_HU
dc.subjectIM - intamuscularhu_HU
dc.subjectiPSCs – induced pluripotent stem cellshu_HU
dc.subjectIV – intravenoushu_HU
dc.subjectKAFOs – knee ankle foot orthoseshu_HU
dc.subjectLBM – lean body masshu_HU
dc.subjectMCK – muscle creatine kinasehu_HU
dc.subjectMDSCs – muscle derived stem cellshu_HU
dc.subjectMMT – manual muscle testinghu_HU
dc.subjectMSCs – mesenchymal stem cellshu_HU
dc.subjectPMO – phosphorodiamidate morpholino oligomerhu_HU
dc.subjectPS – phosphorothioatehu_HU
dc.subjectrhBGN – recombinant human diglycanhu_HU
dc.subjectROM – range of motionhu_HU
dc.subjectROS – reactive-oxygen specieshu_HU
dc.subjectSCs – satellita cellshu_HU
dc.subjectSP cells – side population cellshu_HU
dc.subjectTLRs – troll-like receptorsTMV – tight muscle volumehu_HU
dc.subject.dspaceDEENK Témalista::Orvostudományhu_HU
dc.titlePharmacological treatment of Duchenne and Becker muscular dystrophieshu_HU
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Theses (Faculty of Medicine)