Families of Spinal Muscular Atrophy today announced the funding of an industrial research program at Cambria Bisosciences LLC to identify novel therapeutic targets for Spinal Muscular Atrophy (SMA). Cambria will utilize the model organism Caenorhabditis elegans to mimic genetic aspects of SMA.

“It is now well established that the loss of the SMN1 gene leads to motor neuron loss and muscle degeneration in SMA patients and that the number of SMN2 gene copies modulates disease severity,” said Dr. Beth Westlund of Cambria Biosciences and lead researcher on this project. “Additional studies suggest that other genes can affect the onset and severity of motor neuron loss in this condition. The focus of our research will be to search for genes that can alleviate the problems associated with defects in SMN1 using the roundworm Caenorhabditis elegans or C. elegans, which is a very powerful model organism that is widely used by scientists to identify novel genetic interactions.
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