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Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets
Author
Source
Issue
Vol. 2012, Issue 2012 (31 Dec. 2012), pp.1-13, 13 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2012-04-18
Country of Publication
Egypt
No. of Pages
13
Main Subjects
Abstract EN
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder, leading to progressive muscle weakness, atrophy, and sometimes premature death.
SMA is caused by mutation or deletion of the survival motor neuron-1 (SMN1) gene.
An effective treatment does not presently exist.
Since the severity of the SMA phenotype is inversely correlated with expression levels of SMN, the SMN-encoded protein, SMN is the most important therapeutic target for development of an effective treatment for SMA.
In recent years, numerous SMN independent targets and therapeutic strategies have been demonstrated to have potential roles in SMA treatment.
For example, some neurotrophic, antiapoptotic, and myotrophic factors are able to promote survival of motor neurons or improve muscle strength shown in SMA mouse models or clinical trials.
Plastin-3, cpg15, and a Rho-kinase inhibitor regulate axonal dynamics and might reduce the influences of SMN depletion in disarrangement of neuromuscular junction.
Stem cell transplantation in SMA model mice resulted in improvement of motor behaviors and extension of survival, likely from trophic support.
Although most therapies are still under investigation, these nonclassical treatments might provide an adjunctive method for future SMA therapy.
American Psychological Association (APA)
Tsai, Li-Kai. 2012. Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets. Neural Plasticity،Vol. 2012, no. 2012, pp.1-13.
https://search.emarefa.net/detail/BIM-1002415
Modern Language Association (MLA)
Tsai, Li-Kai. Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets. Neural Plasticity No. 2012 (2012), pp.1-13.
https://search.emarefa.net/detail/BIM-1002415
American Medical Association (AMA)
Tsai, Li-Kai. Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets. Neural Plasticity. 2012. Vol. 2012, no. 2012, pp.1-13.
https://search.emarefa.net/detail/BIM-1002415
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1002415