GENE TRANSFER VECTOR HUMAN CLN2 CDNA BRAIN OF CHILDREN LINCL

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The aim of this study is to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinoses (LINCL), a fatal, autosomal recessive disorder. This will be accomplished by administering to the brain AAV2cuhCLN2, a serotype 2 adenoassociated virus gene transfer vector expressing the normal coding sequences of the CLN2 gene, the gene that is mutated in LINCL.