Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
Shaza S. Issa, Alisa A. Shaimardanova, Valeriya V. Solovyeva, Albert A. Rizvanov
Abstract
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.
Topics & Concepts
Genetic enhancementDiseaseTropismAdeno-associated virusComputational biologyGeneGene deliveryClinical trialBioinformaticsMedicineBiologyVirologyGeneticsVirusVector (molecular biology)PathologyRecombinant DNAVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsCRISPR and Genetic Engineering