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Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a motor neuron disease caused by the loss of survival motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients, which produces low levels of functional protein. Although the SMN2 coding sequence has the potential to produce normal, fu...

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Hlavní autoři: Baughan, Travis D., Dickson, Alexa, Osman, Erkan Y., Lorson, Christian L.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2009
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2667287/
https://ncbi.nlm.nih.gov/pubmed/19228773
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddp076
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