Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy
Dysferlinopathy is a progressive myopathy caused by mutations in the dysferlin (DYSF) gene. Dysferlin protein plays a major role in plasma-membrane resealing. Some patients with DYSF deletion mutations exhibit mild symptoms, suggesting some regions of DYSF can be removed without significantly impact...
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| Main Authors: | , , , , |
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| Format: | Book |
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Elsevier,
2018-12-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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