PATHOGENESIS AND NATURAL HISTORY OF THE GLYCOSPHINGOLIPD (GSL) STORAGE

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。本项目的重点是了解影响中枢神经系统的GSL储存障碍的发病机制，并开发微创方法来监测患有这些疾病的儿童的疾病进展速度和治疗效果。

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 focus of this project is to understand the pathogenesis of GSL storage disorders affecting the central nervous system and to develop minimally invasive methods of monitoring the rate of disease progression and the effectiveness of therapy in children suffering from these disorders.