Delaney Fisher

Keywords adeno-associated virus dependoparvovirus gene therapy capsid engineering neutralizing antibodies immune evasion tissue tropism pre-existing immunity vector redosing Introduction Adeno-associated virus (AAV) is a small, non-enveloped DNA virus commonly used as a gene therapy vector.1,2 The AAV capsid consists of three overlapping proteins—VP1, VP2, and VP3—and packages a 4.7 kilobase genome, flanked by inverted terminal repeats (ITRs).2 The ITRs are the only cis-acting elements...

Abstract BACKGROUND: Cerebral cavernous malformations (CCMs) are vascular neoplasms in the brain that can cause debilitating symptoms. Current treatments pose significant risks to some patients, motivating the development of new nonsurgical options. We recently discovered that focused ultrasound-mediated blood-brain barrier opening (FUS) arrests CCM formation and growth.

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