Journal of Pediatric Epilepsy

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Crystal RG, Sondhi D, Hackett NR , et al.
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.

Hum Gene Ther 2004;
15 (11) 1131-1154

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