Focused Ultrasound-Enhanced CYP46A1 Gene Therapy for Neurodegeneration

h-var-d1632ebb0f

This hybrid approach leverages focused ultrasound with microbubbles to achieve targeted delivery of CYP46A1 overexpression gene therapy directly to affected brain regions in neurodegeneration. The therapeutic mechanism centers on CYP46A1's role as cholesterol 24-hydroxylase, the rate-limiting enzyme for brain cholesterol elimination that converts cholesterol to 24S-hydroxycholesterol. By overexpre