Treatment strategies for spinal muscular atrophy (CROSBI ID 170651)
Prilog u časopisu | pregledni rad (znanstveni) | međunarodna recenzija
Podaci o odgovornosti
Fuller, Heidi ; Barišić, Marija ; Šešo-Šimić, Đurđica ; Špeljko, Tea ; Morris, Glenn E. ; Šimić, Goran
engleski
Treatment strategies for spinal muscular atrophy
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.
clinical trials ; spinal muscular atrophy ; SMN1 gene ; SMN2 gene ; SMN protein ; treatment ; therapy
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Podaci o izdanju
1 (4)
2010.
308-321
objavljeno
2081-3856
2081-6936
10.2478/v10134-010-0045-4
Povezanost rada
Kliničke medicinske znanosti, Psihologija, Temeljne medicinske znanosti