SAG

SAG (Smoothened Agonist) is a potent, cell-permeable small molecule that acts as a direct agonist of the Smoothened (Smo) receptor, a key component of the Hedgehog (Hh) signaling pathway. By binding to the heptahelical bundle of the Smoothened receptor, SAG triggers the activation of downstream Gli transcription factors, effectively mimicking the effects of Sonic Hedgehog (Shh) protein without requiring the Patched (Ptch) receptor. In the context of regenerative medicine and neurobiology, SAG is utilized to promote the differentiation of stem cells, such as human induced pluripotent stem cell-derived neuroepithelial stem cells (hNESCs), into mature neurons. Research, including studies presented at ASGCT, has explored the use of SAG encapsulated in brain-targeted, pH-sensitive liposomes to enhance neuronal engraftment and maturation in the cerebellum, specifically targeting neurodegenerative conditions like Machado-Joseph disease (MJD).

Other names

smoothened agonistSmo Agonist