Recent Developments in Lipid Nanoparticle-Mediated Delivery of Biotherapeutics and Gene Therapy Across the Blood-Brain Barrier
U Iqbal, Rw Hwang, Wj CostainDOI 10.1007/s40259-026-00782-0
Summary
A review of blood–brain barrier transport mechanisms and emerging lipid nanoparticle strategies for carrying nucleic acid and other complex therapies into the central nervous system.
Keywords
Purpose: The blood–brain barrier limits access of many systemically administered therapies to the central nervous system.
Approach: The authors review barrier biology, receptor-mediated transcytosis and recent lipid nanoparticle delivery research; this is not a primary efficacy study.
Key findings: Surface functionalization with barrier-penetrant moieties is being explored to improve CNS delivery of mRNA, siRNA and antisense oligonucleotides. Protein-therapy transport strategies provide a useful comparison, but nanoparticle-mediated delivery still needs validation.
Interpretation: Crossing the barrier remains a central design constraint for CNS gene therapy.
Limitations: Preclinical transport strategies cannot be assumed to translate into safe or effective human treatment.
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