Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

BACKGROUND: 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 n...

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Detalles Bibliográficos
Autores principales: Mattis, Virginia B, Fosso, Marina Y, Chang, Cheng-Wei, Lorson, Christian L
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2009
Materias:
Research article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2789732/
https://www.ncbi.nlm.nih.gov/pubmed/19948047
http://dx.doi.org/10.1186/1471-2202-10-142

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2789732/
https://www.ncbi.nlm.nih.gov/pubmed/19948047
http://dx.doi.org/10.1186/1471-2202-10-142

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