Safety and neuroprotective efficacy of the VCP inhibitor ML240 in large-animal and human retinal explants: a preclinical ex vivo study.

A modest increase in TUNEL labeling was observed at 150 μM; however, it did not reach statistical significance, likely due to the high variability within the control group. mPEG 5kDa -cholane-encapsulated ML240 is well tolerated in monkey and human retinal explants To extend our evaluation to the primate retina, the effects of mPEG 5kDa -cholane-encapsulated ML240 were assessed in retinal explants from adult monkeys and human donors without known disease-causing mutations.

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