Providing new hope to families facing the
debilitating aspects of lysosomal storage diseases.
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Gene Therapy for GM2 Gangliosidosis
Perhaps the greatest opportunity for curing the GM2 Gangliosidosis is the use of gene therapy.
(Tenenbaum 2004, Sands 2006)  The concept has already been demonstrated in multiple small animal
models of lysosomal storage disease (MPSVII - Liu 2005, MPSI - Watson 2006, CLN2 – Passini 2006)
and is currently being used in human clinical investigations for Batten and Canavan diseases.  The
effectiveness of gene therapy in a mouse model of Tay Sachs disease has also already been shown
with the use of a herpes simplex viral vector (Martino 2005) and with the use of an adeno associated
viral (AAV) vector (Cachon-Gonzalez 2006).   While there have been many exciting advances in this
area of gene therapy for the LSD’s, one of the primary challenge for bringing this therapy forward is an
effective means for delivering the vector to broad areas of  brain tissue.  

Go to the Research section to learn more on the gene therapy research being conducted by the New
Hope Research Foundation.