Abstract: Adeno associated viral (AAV) vectors has been used widely in gene therapy and efforts have been made to improve their utility by adding genetic elements that would enable targeting transgene expression to particular cells or tissues of interest and permitting on/off regulation of expression. In this study, we designed a recombinant AAV9 variant PHP.eB vector with muscle creatine kinase (Mck)-derived enhancers, a synthetic muscle-expression promoter, in combination with a third-generation …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 1, Issue 2, 2023 · pp. 46–61 Read article