COMBINATION OF RETROMER PHARMACOLOGICAL CHAPERONES AND EXOGENOUS RETROMER FOR THE TREATMENT OF ALZHEIMER'S DISEASE AND OTHER NEURODEGENERATIVE DISEASES AND DISORDERS

摘要

The present disclosure relates to methods and compositions for elevating and stabilizing retromer for treating and/or preventing Alzheimer's disease and other neurodegenerative diseases or disorders. Additionally, the disclosure relates to gene therapy combined with a pharmacological retromer chaperone therapy for treating and/or preventing Alzheimer's disease (AD), and other neurodegenerative diseases or disorders such as Parkinson's Disease (PD), amyotrophic lateral sclerosis (ALS), neuronal ceroid lipofuscinosis (NCL), and transmissible spongiform encephalopathies (TSEs or prion disease), multiple system atrophy (MSA), as well as tauopathies such as progressive supranuclear palsy (PSP), frontotemporal lobar dementia linked to chromosome 17q21-22 and its subtypes (FTLD- 17/FTLD -Tau), and chronic traumatic encephalopathy (CTE).