Efficacy of prenatal and postnatal antioxidant and neuroprotective ergothioneine supplementation on risdiplam therapy in a SMA mouse model

Department

Department of Pharmaceutical Sciences, University of Milano

Main research activities/topics/projects

Risdiplam is useful in treating Spinal Muscular Atrophy (SMA), partially delaying the death of motor neurons (MNs) and partially restoring the survival motor neuron protein (SMN). The effectiveness of risdiplan decreases as the condition progresses. High levels of oxidative stress are experienced by SMA sufferers, which makes the condition worse. The main goals of this research are to assess how ergothioneine (ERGO) in conjunction with Risdiplam therapy affects the quality of life and survival rate of SMA patients and to look into the mechanisms involving MNs and neural stem cells. In wounded tissue, ERGO is quickly absorbed, strongly retained, and accumulates, where it has potent neuroprotective, cytoprotective, and antioxidant properties. 

SMNΔ7 mice will be used in both an in vitro and an in vivo investigation. The ERGO effect on various muscles, neural stem cells, and spinal cord motor neurons will be assessed.

Working language

Working languages will be English or Italian

Duration in months (min-max)

Master Researcher: 4-12

PhD sandwich: 6-12

Post Doc: 8-12