Intracerebroventricular Administration of Gangliosides for In Vivo Functional Studies in Mice

Levels of gangliosides are reduced in several neurological conditions, and their restoration has shown therapeutic potential in animal models of Huntington's disease and Parkinson's disease. However, the full range of functions of gangliosides in the brain, along with their molecular targets and mechanisms of action following exogenous administration, remains ill-defined. Due to their poor blood-brain barrier (BBB) permeability, gangliosides face significant challenges when delivered through non-invasive routes. Direct intracerebroventricular (ICV) infusion circumvents this issue by bypassing the BBB, ensuring a more precise delivery to brain structures and eliminating uncertainties related to brain penetration. Here, we describe a protocol for ICV infusion of gangliosides in mice that enables in vivo pre-clinical studies of their effects, while eliminating uncertainties about the extent of drug delivery to the brain through the BBB.