Mario Gorziglia

Gene therapy using adenoviral vectors.

Growing interest in adenoviral gene-transfer vectors, stimulated by efforts to develop in vivo gene therapy for cystic fibrosis, has led to an evaluation of their use in many other applications of in vivo gene therapy. Studies are beginning to define strategies for the efficient, albeit transient, expression of the transferred gene and have further identified and partially characterized important host responses to in vivo gene transfer that modulate the duration of expression of the transgene. Ongoing work is actively exploring these issues, with a view to the design of the next generation of adenoviral vectors.

ONCOLYTIC ADENOVIRAL VECTORS

It is important to analyze to what extent these random or designed mutations abrogate viral replication in normal cells because a tightly controlled vector could be injected at higher doses intratumorally or even systemically. On the other hand, it is also important to analyze to what extent these mutations affect the amount of virus produced per infected tumor cell (burst size) compared to wild-type virus because lower yields will result in a slower propagation throughout the tumor. Finally, as concluded from the clinical trials with wildtype.