Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy.

Age-related neurodegenerative diseases have detrimental consequences on health of many patients and result in mortality. The current treatment options are limited and usually fail to correct the underlying pathology. AAV-based gene therapies have proved to be safe based on the data available on clinical trials for several monogenic diseases. Therefore, such therapies can pave the way to treat neurodegenerative diseases likes Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS). Here, the advantages of AAV-based gene therapies are discussed with emphasis on efforts of developing novel capsids with superior therapeutic efficacy. Furthermore, the results of clinical trials on AD, PD, and ALS are summarized.