Brain-targeted delivery of neuroprotective survival gene minimizing hematopoietic cell contamination: implications for Parkinson's disease treatment.
Min Hak LeeSukyeong KangKi-Hwan UmSeok Won LeeHyorin HwangKyunghwa BaekJin Woo ChoiPublished in: Journal of translational medicine (2024)
Our CNS-specific vector system, which encodes a survival transgene DX2, signifies a promising avenue for safe gene therapy, avoiding unintended expression of survival gene in blood cells, applicable to various neurodegenerative diseases.