Adeno-associated virus serotypes for gene therapeutics

L Lisowski, SS Tay, IE Alexander - Current opinion in pharmacology, 2015 - Elsevier
Highlights•A conceptual framework for understanding AAV serotype use in clinical trials.•The
AAV vector toolkit for pseudoserotyping and challenges of clinical translation.•Capsid
engineering technologies to extend the clinical utility of AAV vectors.Gene transfer vectors
based on adeno-associated virus (AAV) are showing exciting therapeutic promise in early
phase clinical trials. The ability to cross-package the prototypic AAV2 vector genome into
different capsids is a powerful way of conferring novel tropism and biology, with evolving …