Spinal muscular atrophy disease: a literature review for therapeutic strategies

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 S...

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Autores principales: Stavarachi, M, Apostol, P, Toma, M, Cimponeriu, D, Gavrila, L
Formato: Texto
Lenguaje:English
Publicado: Carol Davila University Press 2010
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3019038/
https://www.ncbi.nlm.nih.gov/pubmed/20302191
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author Stavarachi, M
Apostol, P
Toma, M
Cimponeriu, D
Gavrila, L
author_facet Stavarachi, M
Apostol, P
Toma, M
Cimponeriu, D
Gavrila, L
author_sort Stavarachi, M
collection PubMed
description 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.
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spelling pubmed-30190382011-03-03 Spinal muscular atrophy disease: a literature review for therapeutic strategies Stavarachi, M Apostol, P Toma, M Cimponeriu, D Gavrila, L J Med Life Review 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. Carol Davila University Press 2010-02-15 2010-02-25 /pmc/articles/PMC3019038/ /pubmed/20302191 Text en ©Carol Davila University Press http://creativecommons.org/licenses/by/2.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Stavarachi, M
Apostol, P
Toma, M
Cimponeriu, D
Gavrila, L
Spinal muscular atrophy disease: a literature review for therapeutic strategies
title Spinal muscular atrophy disease: a literature review for therapeutic strategies
title_full Spinal muscular atrophy disease: a literature review for therapeutic strategies
title_fullStr Spinal muscular atrophy disease: a literature review for therapeutic strategies
title_full_unstemmed Spinal muscular atrophy disease: a literature review for therapeutic strategies
title_short Spinal muscular atrophy disease: a literature review for therapeutic strategies
title_sort spinal muscular atrophy disease: a literature review for therapeutic strategies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3019038/
https://www.ncbi.nlm.nih.gov/pubmed/20302191
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AT cimponeriud spinalmuscularatrophydiseasealiteraturereviewfortherapeuticstrategies
AT gavrilal spinalmuscularatrophydiseasealiteraturereviewfortherapeuticstrategies

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