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A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype

Proximal spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of the survival motor neuron (SMN) protein. In humans, SMN1 and SMN2 encode the SMN protein. In SMA patients, the SMN1 gene is lost and the remaining SMN2 gene only partially compensates. Mediated by a C>T...

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Библиографические подробности
Главные авторы: Gladman, Jordan T., Bebee, Thomas W., Edwards, Chris, Wang, Xueyong, Sahenk, Zarife, Rich, Mark M., Chandler, Dawn S.
Формат: Artigo
Язык:Inglês
Опубликовано: Oxford University Press 2010
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC2951869/
https://ncbi.nlm.nih.gov/pubmed/20705738
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddq343
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