Title |
Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA
|
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Published in |
BMC Neuroscience, November 2009
|
DOI | 10.1186/1471-2202-10-142 |
Pubmed ID | |
Authors |
Virginia B Mattis, Marina Y Fosso, Cheng-Wei Chang, Christian L Lorson |
Abstract |
Spinal Muscular Atrophy (SMA) is the leading genetic cause of infantile death. It is caused by the loss of functional Survival Motor Neuron 1 (SMN1). There is a nearly identical copy gene, SMN2, but it is unable to rescue from disease due to an alternative splicing event that excises a necessary exon (exon 7) from the majority of SMN2-derived transcripts. While SMNDelta7 protein has severely reduced functionality, the exon 7 sequences may not be specifically required for all activities. Therefore, aminoglycoside antibiotics previously shown to suppress stop codon recognition and promote translation read-through have been examined to increase the length of the SMNDelta7 C-terminus. |
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Geographical breakdown
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Unknown | 33 | 94% |
Demographic breakdown
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Student > Master | 3 | 9% |
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