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.
Keyphrases
- gene therapy
- free survival
- induced apoptosis
- copy number
- poor prognosis
- cerebral ischemia
- single cell
- blood brain barrier
- cell therapy
- drinking water
- multiple sclerosis
- cell cycle arrest
- gene expression
- cell proliferation
- oxidative stress
- endoplasmic reticulum stress
- genome wide identification
- binding protein
- human health
- genome wide analysis
- functional connectivity