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A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy

Spinal muscular atrophy, the leading genetic killer of infants, is caused by reduced levels of SMN protein. Drugs that increase SMN might convert severe babies into milder children, or allow children to survive as adults. This work describes the development of an ELISA-based assay for the identification of drugs that increase SMN protein in cell models. A review of the work in Neurology Today quotes “this small study is encouraging”….“Previous tests of the SMN protein were not sufficiently specific and sensitive” and “this work paves the way for a more sensitive and specific ELISA assay.” (http://journals.lww.com/neurotodayonline/Fulltext/2008/12040/New_ELISA_Assay_May_Enable_More_Sensitive_Testing.7.aspx).