METHODS AND COMPOSITIONS FOR TREATMENT OF FRONTOTEMPORAL DEMENTIA

摘要

Disclosed are methods and compositions for treating cognitive impairment due to frontotemporal dementia caused by progranulin mutations. More specifically, the invention relates to antisense oligonucleotides (ASOs) and their administration to subjects to increase progranulin protein levels in brain tissue, to treat cognitive impairment due to frontotemporal dementia caused by progranulin mutations.