Provided are compositions and methods for treating early onset familial Alzheimer's Disease. The compositions and methods can be used for improving one or more aspects of cognitive function in a patient suffering from early onset familial Alzheimer's Disease. In certain embodiments, the patient has autosomal dominant early-onset Alzheimer's Disease. In some embodiments, the patient has a mutation in at least one gene selected from the group consisting of PSEN 1, PSEN2 and APP. In some embodiments, the PSEN 1 mutation is a change or deletion in one or more amino acids. In some embodiments, the patient has a mutation in PSEN 1 (e.g., a missense mutation).
提供了用于治疗早发家族性阿尔茨海默病的组合物和方法。这些组合物和方法可用于改善患有早发家族性阿尔茨海默病患者的一个或多个认知功能方面。在某些实施例中,患者患有常染色体显性早发阿尔茨海默病。在某些实施例中,患者至少有一种来自P
SEN 1、P
SEN2和APP
基因组合中的
基因突变。在某些实施例中,P
SEN 1
基因突变是一个或多个
氨基酸的改变或缺失。在某些实施例中,患者患有P
SEN 1
基因突变(例如,错义突变)。