Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Inclusion Criteria: * A definitive diagnosis of late infantile neuronal ceroid lipofuscinosis, based on clinical phenotype and genotype, with CLN2 gene mutations known to be associated with the disease. * All subjects will be naive, i.e., they have not previously participated in a gene therapy study for LINCL. * Parents of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments. * Both parents or legal guardians must give consent for their child's participation in the research study. * For group A, subjects will have a LINCL average total disability score 0 to 4, the severe form of the disease. * For group B, subjects will have a LINCL average total disability score 5 to 6, a moderate form of the disease. Exclusion criteria * Other significant medical or neurological conditions may disqualify the patient from participation in this study, particularly those which would create an unacceptable operative risk or risk to receiving the AAV2CUhCLN2 vector. Examples include malignancy (other than skin cancer), congenital heart disease, liver or renal failure, or seropositive for HIV. Each case will be individually reviewed and the final decision shall rest with the Eligibility Committee comprised on three physicians other than the Principal Investigator, including a pediatric neurosurgeon, pediatric neurologist and general pediatrician. * Individuals without adequate control …