A new generation of SMA therapeutics designed to increase levels of SMN protein in various tissues are currently being developed. Preclinical and clinical testing of many of these potential therapeutics requires a sensitive and reliable assay for measuring SMN levels in cells and tissue before and after treatment. The SMA Foundation, in collaboration with Assay Designs/Enzo Life Sciences, has developed a sensitive ELISA (Enzyme-Linked Immunosorbent Assay) kit for the quantitative determination of SMN protein levels. The ELISA kit is manufactured using standardized processes to maximize accuracy and reproducibility of results and can measure SMN protein in samples with concentrations as low as 50pg/mL. The assay was validated using human peripheral blood mononuclear cells (PBMCs), and also can be used to measure SMN in other tissues like skin fibroblasts and muscle that are accessible for collection*. The assay can also be used to detect SMN protein in various mouse tissues, including brain and spinal cord tissues. Protocols for measuring SMN levels in these tissues are available. The kit is available for purchase from Enzo Life Sciences.
*SMN protein was undetectable in healthy human cerebrospinal fluid (CSF), saliva, buccal cells or urine using the ELISA.