Whole cell panning;Identify Tuberous Sclerosis Markers

描述（由申请人提供）：我们描述了人肾血管平滑肌脂肪瘤（AML）细胞系的发展，该细胞系提供了用于鉴定结节性硬化症（TSC）分子标志物的体外细胞模型。具体来说，该提议的一个目标是使人类AML细胞永生化，并产生一组匹配的TSC基因敲入控制细胞系。我们将使用这些细胞系试剂使用幼稚噬菌体M13抗体文库进行全细胞生物筛选。AML特异性标志物将使用受tsc影响的组织样本和正常组织样本进行验证。这一建议将形成一系列研究的基础，以确定由TSC1或TSC2基因失活导致的细胞标记物。目前治疗TSC的方法是手术切除由疾病引起的病变。此选项不适用于某些临床表现。我们研究的最终目标是获得一种用于TSC免疫治疗的人单克隆抗体，可用于所有TSC患者。

DESCRIPTION (provided by applicant): We describe the development of human renal angiomyolipoma (AML) cell lines with which to provide an in vitro cellular model for use in identifying molecular markers of Tuberous Sclerosis Complex (TSC). Specifically, one goal of this proposal is to immortalize human AML cells and generate a set of matching TSC gene knock-in control cell lines. We shall use these cell-line reagents for whole cell biopanning using a naive bacteriophage M13 antibody library. AML specific markers will be validated using TSC-affected, and normal tissue samples. This proposal will form the foundation to a series of studies to identify cell markers that result from the inactivation of either the TSC1 or TSC2 genes. The current therapy for TSC is surgery to remove the lesions that result as a consequence of the disease. This option is not available for some clinical manifestations. The ultimate goal of our studies will be to derive a human monoclonal antibody for the immunotherapeutic treatment of TSC that can be used for all TSC patients.