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Gene Therapy for Late Infantile Neuronal Ceroid Lipofuscinoses (LINCL)

Detailed Technology Description

Invention SummaryThis invention allows for the treatment of LINCL by intracranial administration of an adeno-associated virus (AAV) carrying the cDNA for the CLN2 gene.

Others

Publications

· Hackett N.R., et. al., Human Gene Therapy16:1484-1503 (2005)

· Clinical Protocol: Human Gene Therapy15:1131-1154 (2004)

Country/Region

USA

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