GENE TRANSFER VECTOR HUMAN CLN2 CDNA BRAIN OF CHILDREN LINCL

基因转移载体 人类 CLN2 CDNA 儿童大脑 LINCL

• 批准号: 7378367
• 负责人: RONALD G CRYSTAL
• 金额: $ 20.26万
• 依托单位: WEILL MEDICAL COLL OF CORNELL UNIV
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7378367
• 关键词: GENE; TRANSFER; VECTOR; HUMAN; CLN2

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.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。本研究的目的是治疗晚期婴儿神经元蜡样脂褐质沉积症（LINCL）的中枢神经系统（CNS）表现，LINCL是一种致命的常染色体隐性遗传疾病。这将通过向大脑施用AAV 2cuhCLN 2来实现，这是一种血清型2型腺相关病毒基因转移载体，表达CLN 2基因（LINCL中突变的基因）的正常编码序列。