R. Jude Samulski, PhD
and members of the Gene Therapy Center have recently developed
novel viral vectors with targeting capability. These reagents
will be of significant value when transducing specific cell
types such as hepatocytes.
of the great aspirations of gene therapy is to eventually
develop technology which will provide a feasible approach
to correcting genetic defects and combating infectious diseases.
The laboratory is engaged in studying the molecular biology
of the defective human parvovirus adeno-associated virus (AAV)
in hopes of developing a safe, efficient viral vector for
human gene therapy. Ongoing research is revealing that this
nonpathogenic human virus is now accessible for utilization
as a vector. We have developed a packaging system which allows
for efficient encapsidation of foreign genes into AAV virions.
We have also indentified the essential cis-acting
sequencing required for efficient integration into host cell
DNA. Finally, we have characterized this integration step
and uncovered the exciting result of site-specific integration.
this last observation clearly sets AAV apart as a eucaryotic
viral vector and its potential for gene therapy in humans.
Our continued efforts to understand and manipulate this virus
as a vector has required a multi-faceted approach from basic
virology in vivo to in vitro virus replication,
integration, and packaging.
||Ping Jie Xiao