KUS121 is a valosin-containing protein modulator that inhibits VCP ATPase activity with an IC50 of 330 nM, suppresses tunicamycin-induced ATP depletion, cell death, and CHOP upregulation in HeLa cells, protects primary cortical neurons from oxygen-glucose deprivation injury in vitro, reduces infarct volume and improves motor performance in mouse models of focal cerebral ischemia, and preserves retinal structure and visual function in rd10 retinitis pigmentosa models, supporting its investigation in neuroprotection and ischemic disease research.

In animal models of ischemic retinal injury, intravitreal (IVT) injection of KUS121 (25 ug/eye) mitigates retinal damage. The administration is associated with the suppression of inner retinal layer thinning at 7 and 14 days post-injury, preserving retinal morphology and visual function. In rd10 mice (a model for retinitis pigmentosa), KUS121 inhibits the thinning of the outer nuclear layer and maintains visual acuity. These in vivo effects are linked to the suppression of Endoplasmic Reticulum (ER) stress, characterized by reduced expression of the marker CHOP, activation of the mTOR signaling pathway, and inhibition of apoptosis [1][2][3][4].

DMSO: 48 mg/mL (104.7 mM), Sonication is recommended.