A comparison of AAV-vector production methods for gene therapy and preclinical assessment.

ABSTRACT: Adeno Associated Virus (AAV)-mediated gene expression in the brain is widely applied in the preclinical setting to investigate the therapeutic potential of specific molecular targets, characterize various cellular functions, and model central nervous system (CNS) diseases. In therapeutic applications in the clinical setting, gene therapy offers several advantages over traditional pharmacological based therapies, including the ability to directly manipulate disease mechanisms, selectively target disease-afflicted regions, and achieve long-term therapeutic protein expression in the absence of repeated administration of pharmacological agents. Next to the gold-standard iodixanol-based AAV vector production, we recently published a protocol for AAV production based on chloroform-precipitation,

SUBMITTER: Davidsson M 

PROVIDER: S-EPMC7726153 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature