The blood-brain barrier (BBB) prevents neurotoxic plasma components, blood cells, and
pathogens from entering the brain. At the same time, the BBB regulates transport of …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

The monogenic nature of Huntington's disease (HD) and other neurodegenerative diseases
caused by the expansion of glutamine-encoding CAG repeats makes them particularly …

Widespread distribution of gene products at clinically relevant levels throughout the CNS
has been challenging. Adeno-associated virus type 9 (AAV9) vector has been reported as a …

Some recombinant adeno-associated viruses (rAAVs) can cross the neonatal blood–brain
barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However …

The presence of the blood-brain barrier (BBB) remains a challenge in the treatment of
central nervous system (CNS) diseases, as it hinders the infiltration of many therapeutic …

Noninvasive drug delivery to the brain remains a major challenge for the treatment of
neurological disorders. Transcranial focused ultrasound combined with lipid-coated gas …

Lysosomal Storage Diseases are multisystemic disorders determined by genetic variants,
which affect the proteins involved in lysosomal function and cellular metabolism. Different …

Global gene delivery to the CNS has therapeutic importance for the treatment of
neurological disorders that affect the entire CNS. Due to direct contact with the CNS …

The ability to precisely upregulate genes in inflamed brain holds great therapeutic promise.
Here we report a novel class of vectors, genetically modified macrophages that carry …