CYP46A1 Small Molecule Activator Therapy

h-var-32c4875adb

This approach employs pharmacological activation of endogenous CYP46A1 rather than gene overexpression to treat Alzheimer's disease through enhanced brain cholesterol metabolism. Small molecule activators specifically target the CYP46A1 enzyme's allosteric binding sites, increasing catalytic efficiency by 3-5 fold without altering protein expression levels. The lead compound, CYP46-ACT1, demonstra