CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND USES IN GENE THERAPY OF HUMAN LIVER CANCER

摘要

The present invention discloses a new generation of multiple mutated capsid protein-modified rAAV expression vectors, and also infectious virions, and compositions and pharmaceutical formulations comprising the same. In addition, the present invention provides a method for preparing these highly efficient transduction vector constructs and for various therapeutic applications, particularly in vivo and/or ex situ viral vector-based gene therapy protocols in one or more diseases or abnormal conditions in diseased mammals. As a delivery agent to treat or ameliorate. In addition, the present invention relates to a method for large-scale production of multiple mutated capsid-modified rAAV expression vectors, viral particles, and infectious virions, and also in the manufacture of a medicament for use in various in vitro and/or in vivo treatment methods. Disclosed is the use of the disclosed compositions.