Gene therapy: a primer for neurosurgeons
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Updated: Aug 13, 2026

Intraventricular Transplantation of Engineered Neuronal Precursors for In Vivo Neuroarchitecture Studies
Published on: May 11, 2019
1Molecular Neuro-oncology Laboratory, Neurosurgery Service, Massachusetts General Hospital, Brain Tumor Center, Boston, Massachusetts 02114, USA. Chiocca@helix.mgh.harvard.edu
Abstract:
Gene therapy involves the transfer of genes into cells with therapeutic intent. Although several methods can accomplish this, vectors based on viruses still provide the most efficient approach. For neurosurgical purposes, preclinical and clinical applications in the areas of glioma therapy, spinal neurosurgery, and neuroprotection for treatment of Parkinson's disease and cerebral ischemia are reviewed. In general, therapies applied in the neurosurgical realm have proven relatively safe, despite occasional, well-publicized cases of morbidity and death in non-neurosurgical trials. However, continued clinical and preclinical research in this area is critical, to fully elucidate potential toxicities and to generate truly effective treatments that can be applied in neurological diseases.

