Servio H Ramirez, Jonathan Hale, Siobhan McCarthy, Christian L Lino Cardenas, Kalpani Dona, Killian S Hanlon, Eloise Hudry, Demitri De La Cruz, Carrie Ng, Sabyasachi Das, Diane M Nguyen, Josette Nammour, Rachel E Bennett, Allison Andrews, Patricia L Musolino, Casey A Maguire
Neurodegeneration and cerebrovascular disease share an underlying microvascular dysfunction that may be remedied by selective transgene delivery. To date, limited options exist in which cellular components of the brain vasculature can be effectively targeted by viral vector therapeutics. Here, we characterize the first engineered adeno-associated virus (AAV) capsid mediating high transduction of cerebral vascular pericytes and smooth muscle cells. We performed two rounds of in vivo selection with an AAV capsid scaffold displaying a heptamer peptide library to isolate capsids that traffic to the brain after intravenous (iv) delivery...
June 27, 2023: Human Gene Therapy