JNK INHIBITORS FOR USE IN TREATING SPINAL MUSCULAR ATROPHY

摘要

The brain specific isoform (JNK3) of c-Jun NH2-terminal kinase (JNK) has been found to mediate the degeneration of spinal motor neurons caused by SMN deficiency in spinal muscular atrophy (SMA). Moreover, the ability of JNK inhibitors to reduce degeneration of neurons lacking SMN is also disclosed. The JNK signaling pathway can therefore mediate neurodegeneration in SMA and represents a therapeutic target for treatment of SMA.