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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Bibliographic Details
Published inJournal of medicine and life Vol. 3; no. 1; pp. 3 - 9
Main Authors Stavarachi, M, Apostol, P, Toma, M, Cimponeriu, D, Gavrila, L
Format Journal Article
LanguageEnglish
Published Romania Carol Daila University Foundation 2010
Carol Davila University Press
Subjects
Anabolic Agents - therapeutic use
Exons
Genetic Therapy
Humans
Muscular Atrophy, Spinal - genetics
Muscular Atrophy, Spinal - therapy
Neuroprotective Agents - therapeutic use
Promoter Regions, Genetic
Review
Stem Cell Transplantation
Survival of Motor Neuron 2 Protein - genetics
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Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
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ObjectType-Review-1
ISSN:1844-122X
1844-3117