Behavioral and Brain Sciences

Open Peer Commentary

Multiple obstacles to gene therapy in the brain

David Avram Sandersa1

a1 Whitehead Institute for Biomedical Research, Nine Cambridge Center, Cambridge, MA 02142.


Neuwelt et al. have proposed gene-transfer experiments utilizing an animal model that offers many important advantages for investigating the feasibility of gene therapy in the human brain. A variety of tissues concerning the viral vector and mode of delivery of the corrective genes need to be resolved, however, before such therapy is scientifically supportable.