Narrative review demonstrates that focused ultrasound with microbubbles enables reversible blood–brain barrier opening for CNS gene delivery, highlighting its therapeutic potential.
Key Points
Synthesize preclinical and clinical evidence on focused ultrasound combined with microbubbles to transiently disrupt the blood–brain barrier for targeted central nervous system therapeutic delivery.
Reviewed preclinical animal models and early-phase clinical trials evaluating blood–brain barrier microdisruption across multiple neurological conditions.
Assessed technical parameters including acoustic settings, microbubble properties, and real-time cavitation monitoring systems.
Preclinical studies across Parkinson’s, Alzheimer’s, Huntington’s, glioblastoma, and ALS demonstrated enhanced gene delivery, increased transgene expression, and improved functional outcomes.
Large-animal and early human studies confirmed that barrier opening is reversible, spatially localized, and generally well tolerated with measurable improvements in drug penetration.
Real-time cavitation monitoring, acoustic tuning, and microbubble optimization were identified as critical factors for ensuring safety and therapeutic efficacy.