Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches

Viruses. 2020 Apr 18;12(4):460. doi: 10.3390/v12040460.


Neurodegenerative diseases (NDDs) are most commonly found in adults and remain essentially incurable. Gene therapy using AAV vectors is a rapidly-growing field of experimental medicine that holds promise for the treatment of NDDs. To date, the delivery of a therapeutic gene into target cells via AAV represents a major obstacle in the field. Ideally, transgenes should be delivered into the target cells specifically and efficiently, while promiscuous or off-target gene delivery should be minimized to avoid toxicity. In the pursuit of an ideal vehicle for NDD gene therapy, a broad variety of vector systems have been explored. Here we specifically outline the advantages of adeno-associated virus (AAV)-based vector systems for NDD therapy application. In contrast to many reviews on NDDs that can be found in the literature, this review is rather focused on AAV vector selection and their preclinical testing in experimental and preclinical NDD models. Preclinical and in vitro data reveal the strong potential of AAV for NDD-related diagnostics and therapeutic strategies.

Keywords: AAV; gene therapy; neuro-degenerative disease.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Central Nervous System / metabolism
  • Central Nervous System / pathology
  • Dependovirus / genetics*
  • Disease Models, Animal
  • Environment
  • Gene Targeting
  • Gene Transfer Techniques
  • Genetic Therapy* / adverse effects
  • Genetic Therapy* / methods
  • Genetic Vectors / genetics*
  • Humans
  • Neurodegenerative Diseases / diagnosis
  • Neurodegenerative Diseases / etiology
  • Neurodegenerative Diseases / therapy*
  • Transgenes