337. Transient Ultrasound-Mediated Microbubble-Assisted Modulation of Blood-Brain Interface in Adult Common Marmoset to Improve rAAV-Mediated Brain Transduction

Background: Non-human primates (NHPs) could provide an appropriate model for neuromuscular diseases because of its cognitive function and physiological resemblance to human. Production of transgenic and knockout NHPs from preimplantation embryos by using genomic modification were reported. However, several decades would be required for establishing homozygous and/or phenotypically stable progenies and for developing the symptoms in case of late...