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.
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H. Voss | M. Souweidane | P. Stieg | D. Ballon | R. Crystal | J. Dyke | L. Heier | N. Hackett | M. Kaplitt | K. Wisniewski | S. Worgall | B. Greenwald | Joy Howell | M. Dinner | S. Kaminsky | S. Hosain | Kristin Strybing | D. Sondhi