Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

The glymphatic system is a brain-wide clearance pathway; its impairment contributes to the
accumulation of amyloid-β. Influx of cerebrospinal fluid (CSF) depends upon the expression …

Key points It is unclear precisely how macromolecules (eg endogenous proteins and
exogenous immunotherapeutics) access brain tissue from the cerebrospinal fluid (CSF). We …

Attention-deficit hyperactivity disorder (ADHD) is a neurodevelopmental disorder
characterised by developmentally inappropriate levels of inattention and hyperactivity or …

Convection-enhanced delivery (CED) is a promising technique that generates a pressure
gradient at the tip of an infusion catheter to deliver therapeutics directly through the …

Targeted therapies for cancer is a rapidly advancing field, but the identification of tumor-
specific ligands has proven difficult. Chlorotoxin (CTX) is a small, 36 amino acid neurotoxin …

The detection of myelin disruptions in Alzheimer's disease (AD)–affected brain raises the
possibility that oligodendrocytes undergo pathophysiological assault over the protracted …

Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …

Glioblastoma multiforme (GBM) is one of the most frequently occurring tumors in the central
nervous system and the most malignant tumor among gliomas. Despite aggressive …