Spinal muscular atrophy disease: a literature review for therapeutic strategies

Autor:	Stavarachi, M, Apostol, P, Toma, M, Cimponeriu, D, Gavrila, L
Jazyk:	angličtina
Rok vydání:	2010
Předmět:	Review Exons Genetic Therapy mutations neuromuscular disorder nervous system diseases Muscular Atrophy Spinal Survival of Motor Neuron 2 Protein therapeutically drugs Anabolic Agents Neuroprotective Agents SMN gene Humans Promoter Regions Genetic Stem Cell Transplantation
Zdroj:	Journal of Medicine and Life
ISSN:	1844-3117 1844-122X
Popis:	Currently, there is no cure for the treatment of spinal muscular atrophy (SMA). Based on the available clinical and molecular findings, different therapeutic strategies were tested in vitro and in vivo and clinical trials are ongoing. The main therapeutic direction is focused on the enhancement of SMN expression by increasing the full-length (fl) SMN2 transcript levels, preventing the SMN exon 7 from skipping or from protein stabilizing. In addition, the action of neurotrophic, neuroprotective or anabolic agents is tested and stem cell and gene therapy approaches are in a promising development.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=pmid________::d862b9075206ff496eeeaf572d2cf296 http://europepmc.org/articles/PMC3019038 Zobrazit plný text záznamu