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Pregled bibliografske jedinice broj: 507779

Časopis

Autori: Fuller, Heidi; Barišić, Marija; Šešo-Šimić, Đurđica; Špeljko, Tea; Morris, Glenn E.; Šimić, Goran
Naslov: Treatment strategies for spinal muscular atrophy
Izvornik: Translational Neuroscience (2081-3856) 1 (2010), 4; 308-321
Vrsta rada: pregledni rad
Ključne riječi: clinical trials; spinal muscular atrophy; SMN1 gene; SMN2 gene; SMN protein; treatment; therapy
Sažetak:
Progress in understanding the genetic basis and pathophysiology of spinal muscular atrophy (SMA), along with continuous efforts in finding a way to increase survival motor neuron (SMN) protein levels have resulted in several strategies that have been proposed as potential directions for efficient drug development. Here we provide an overview on the current status of the following approaches: 1) activation of SMN2 gene and increasing full length SMN2 transcript level, 2) modulating SMN2 splicing, 3) stabilizing SMN mRNA and SMN protein, 4) development of neurotrophic, neuroprotective and anabolic compounds and 5) stem cell and gene therapy. The new preclinical advances warrant a cautious optimism for emergence of an effective treatment in the very near future.
Projekt / tema: 108-1081870-1942
Izvorni jezik: ENG
Current Contents: NE
Citation Index: NE
Kategorija: Znanstveni
Znanstvena područja:
Temeljne medicinske znanosti,Kliničke medicinske znanosti,Psihologija
Tiskani medij: da
URL Internet adrese: http://www.springerlink.com/content/2561428n145523p1/
Broj citata:
Altmetric:
DOI: 10.2478/v10134-010-0045-4
Google Scholar: Treatment strategies for spinal muscular atrophy
Upisao u CROSBI: gsimic@mef.hr (gsimic@mef.hr), 28. Ožu. 2011. u 11:49 sati



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