Brain-penetrant AAV capsid progress at ASGCT
Companies and academics showcase two approaches to make AAV capsids with enhanced brain targeting and suppressed liver targeting
Gene therapies could transform treatment of many neurological diseases, if the viral vectors were better at getting into the brain. At this year's ASGCT meeting, at least a dozen research teams presented progress in engineering capsids to cross the blood-brain barrier.
Whether by delivering a functional copy of a mutant gene, or delivering a DNA-encoded antibody or protein therapy, AAV-mediated gene therapy promises new solutions to both rare and common CNS diseases. ...