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Nat Methods. 2010 Nov;7(11):905-7. doi: 10.1038/nmeth.1518. Epub 2010 Oct 17.

Efficient CNS gene delivery by intravenous injection.

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  • 1Department of Pathology, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.


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.

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