Various AAV Serotypes and Their Applications in Gene Therapy: An Overview.
Shaza S IssaAlisa A ShaimardanovaValeriya Vladimirovna SolovyevaAlbert Anatolyevich RizvanovPublished in: Cells (2023)
Despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Today, many AAV-based gene therapy medications are being investigated in preclinical and clinical trials, and new ones are appearing on the market. In this article, we present a review of AAV discovery, properties, different serotypes, and tropism, and a following detailed explanation of their uses in gene therapy for disease of different organs and systems.
Keyphrases
- gene therapy
- cell therapy
- genome wide
- copy number
- clinical trial
- stem cells
- mesenchymal stem cells
- small molecule
- genome wide identification
- dna methylation
- health insurance
- randomized controlled trial
- high throughput
- transcription factor
- gene expression
- study protocol
- mass spectrometry
- single molecule
- genetic diversity