Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
The adeno-associated virus (AAV) is one of the most potent vectors in gene therapy. The experimental profile of this vector shows its efficiency and accepted safety, which explains its increased usage by scientists for the research and treatment of a wide range of diseases. These studies require using functional, pure, and high titers of vector particles. In fact, the current knowledge of AAV structure and genome helps improve the scalable production of AAV vectors. In this review, we summarize the latest studies on the optimization of scalable AAV production through modifying the AAV genome or biological processes inside the cell.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10121408 | PMC |
| http://dx.doi.org/10.1016/j.heliyon.2023.e15071 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
AAV2 delivery of the gene results in presentation of an HLA-A02:01-restricted T cell epitope potent to induce T cell cytotoxicity.
Mol Ther Methods Clin Dev
September 2025
Office of Gene Therapy, Office of Therapeutic Products, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
genome editing with CRISPR-Cas9 systems is generating worldwide attention and enthusiasm for the possible treatment of genetic disorders. However, the consequences of potential immunogenicity of the bacterial Cas9 protein and the AAV capsid have been the subject of considerable debate. Here, we model the antigen presentation in cells after gene editing by transduction of a human cell line with an AAV2 vector that delivers the Cas9 transgene.
View Article and Find Full Text PDFReply to 'Inborn errors of immunity and AAV: a complex picture'.
Nat Rev Rheumatol
September 2025
Department of Pediatric Rheumatology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Tropism and Retinal Transduction Efficiency of Adeno-Associated Virus Serotypes in Mice.
Invest Ophthalmol Vis Sci
September 2025
Department of Ophthalmology, Dean McGee Eye Institute, University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States.
Purpose: Adeno-associated viruses (AAVs) have become the preferred vector for gene therapy in ophthalmology. However, requirements for specific cell surface receptors limit AAV-mediated retinal cell transduction efficiency. This led to the need to engineer novel AAV vectors for widespread retinal transduction and transgene expression.
View Article and Find Full Text PDFInnate immune response to AAV-based gene therapy vectors: Mechanisms of complement activation and cytokine release.
Mol Ther Methods Clin Dev
September 2025
Roche Pharma Research and Early Development, Roche Innovation Center Basel, 4070 Basel, Switzerland.
Recombinant adeno-associated viruses (rAAV) have emerged as a preferred strategy for gene delivery. However, the immune response to rAAV presents a major limitation, leading to serious adverse events in clinical trials. This study investigates the interaction between rAAV and the innate immune system.
View Article and Find Full Text PDFA Comparative Study of Transfection Techniques for Genetic Modification in Chicken Primordial Germ Cells.
Mol Biotechnol
September 2025
Shandong Provincial Key Laboratory for Livestock Germplasm Innovation & Utilization, College of Animal Science, Shandong Agricultural University, Taian, China.
Avian species possess a distinct reproductive system that differs fundamentally from mammals, resulting in relatively slower progress in transgenic poultry research compared to mammalian models. Primordial germ cells (PGCs), as the precursors to functional gametes, provide a promising avenue for the utilization of genetic resources and the application of transgenesis in chickens. However, the transfection of chicken PGCs remains challenging due to the low efficiency of conventional methods.
View Article and Find Full Text PDF