C12Y304/14008

TREATMENT OF LYSOSOMAL STORAGE DISEASE IN THE EYE THROUGH ADMINISTRATION OF AAVS EXPRESSING TPP1

Provided are methods of treating the retinal dysfunction in a mammal with lysosomal storage disorder which method comprises sub-retinal administration of recombinant AAV particles encoding a soluble lysosomal tripeptidyl peptidase 1 (TPP1). In particular, the retinal dysfunction may be occurring in children with CLN2 deficiency receiving enzyme replacement therapy or gene therapy for their disease.