Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

Crossing the blood–brain barrier in primates is a major obstacle for gene delivery to the
brain. Adeno-associated viruses (AAVs) promise robust, non-invasive gene delivery from the …

Simple Summary Adeno-associated virus (AAV) is a small, non-pathogenic, and replication-
defective virus that mainly infects primates. AAV has demonstrated great success in pre …

Viruses have evolved the ability to bind and enter cells through interactions with a wide
variety of cell macromolecules. We engineered peptide-modified adeno-associated virus …

Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by
mutations in the SMN1 gene. Despite the development of various therapies, outcomes can …

Angelman syndrome (AS) is a neurogenetic disorder caused by the loss of ubiquitin ligase
E3A (UBE3A) gene expression in the brain. The UBE3A gene is paternally imprinted in brain …

Recombinant adeno-associated viral vectors (rAAVs) are a promising strategy to treat
neurodegenerative diseases because of their ability to infect non-dividing cells and confer …

Many diseases affecting the central nervous system (CNS) are deadly but less understood,
leading to impaired mental and motor capabilities and poor patient prospects. Gene therapy …

Introduction The blood–brain barrier (BBB) restricts brain access of virtually all
macromolecules. Receptor-mediated transcytosis (RMT) is one strategy toward their brain …