No evidence for tumorigenesis of AAV vectors in a large-scale study in mice.

Six hundred ninety-five mice received adeno-associated virus (AAV) vectors, mostly via portal vein injection. At necropsy, the livers were inspected for tumors, and tissue sections were prepared for histology. We observed only one tumor, a lipoma, resulting in a tumor frequency of 0.

An optimized protocol for detection of E. coli beta-galactosidase in lung tissue following gene transfer.

Staining by 5-bromo-4-chloro-3-indolyl-beta-D: -galactopyranoside (X-gal) typically detects activity of E. coli beta-galactosidase (beta-gal) in transduced tissues that express the LacZ reporter gene. In lung tissue from mice that received beta-galactosidase-expressing adeno-associated virus (AAV) vectors via intranasal inhalation, we observed only a low frequency of positive cells after X-gal staining in contrast to other reporter genes, such as alkaline phosphatase or green fluorescent protein.