Scientists have developed novel tools and methods for delivering therapeutic genes to cells in the central nervous system that hold great promise for the development of new treatments to treat brain tumors and other neurological disorders.
They have reported the successful use of stem cells derived from human umbilical cord blood to deliver a novel therapeutic gene-interleukin-12-to the brain for long-term anti-tumor activity against gliomas, a deadly type of brain tumor.
Their study highlights the latest genetic tools that are enabling cell type-specific delivery of transgenes for studying the structure and function of neuronal circuits.
"Treating neurologic diseases with traditional biologic products such as therapeutic proteins has been challenging due to limited access. The use of vectors helps to overcome these barriers," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
The study was published in the journal Human Gene Therapy.