Related Experiment Video
Updated: Jun 8, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
Published on: May 16, 2025
Efficient CNS gene delivery by intravenous injection
Jean-Pierre Louboutin1, Alena A Chekmasova, Elena Marusich
1Department of Pathology, Thomas Jefferson University, Philadelphia, Pennsylvania, USA. jplouboutin@hotmail.com
Abstract:
We administered recombinant SV40-derived viral vectors (rSV40s) intravenously to mice with or without prior intraperitoneal injection of mannitol to deliver transgenes to the central nervous system (CNS). We detected transgene-expressing cells (mainly neurons) most prominently in the cortex and spinal cord; prior intraperitoneal mannitol injection increased CNS gene delivery tenfold. Intravenous injection of rSV40s, particularly with mannitol pretreatment, resulted in extensive expression of multiple transgenes throughout the CNS.

