Behavioral and Brain Sciences

Open Peer Commentary

The limitations of central nervous systemdirected gene transfer

Beverly L. Davidsona1

a1 Department of Internal Medicine, University of Michigan Medical Center, Ann Arbor, MI 48109-0680.


Complementation and correction of a genetic defect with CNS manifestations lags behind gene therapy for inherited disorders affecting other organ systems because of shortcomings in delivery vehicles and access to the CNS. The effects of improvements in viral and nonviral vectors, coupled with the development of delivery strategies designed to transfer genetic material thoughout the CNS are being investigated by a number of laboratories in efforts to overcome these problems.