GENE TRANSFER VECTOR HUMAN CLN2 CDNA BRAIN OF CHILDREN LINCL

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。本研究的目的是治疗晚期婴儿神经性ceroid脂褐质病（LINCL）的中枢神经系统（CNS）表现，这是一种致命的常染色体隐性遗传病。这将通过给予脑AAV2cuhCLN2来完成，AAV2cuhCLN2是一种血清2型腺相关病毒基因转移载体，表达CLN2基因的正常编码序列，该基因在LINCL中发生突变。

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.