Dogbey Dennis Makafui, Barth Stefan
Medical Biotechnology and Immunotherapy Research Unit, Institute of Infectious Disease and Molecular Medicine, Faculty of Health Sciences, University of Cape Town, Cape Town, 7700, South Africa.
South African Research Chair in Cancer Biotechnology, Department of Integrative Biomedical Sciences, Faculty of Health Sciences, University of Cape Town, Cape Town, 7700, South Africa.
Mol Biotechnol. 2025 Jan 29. doi: 10.1007/s12033-025-01381-0.
The field of gene therapy has witnessed significant advancements in the utilization of Adeno-associated virus (AAV) owing to its inherent biological advantages. Targeted AAV vectors are generated through genetic or chemical modification of the capsid for user-directed purposes. However, this process can result in imbalances in viral protein sequence homogeneity, stoichiometry, and functional transduction vector units, thereby introducing new challenges. This mini review focuses on the ongoing efforts to develop targeted vectors, which inadvertently present unsolicited obstacles for clinical application and provided perspectives on future directions.
由于腺相关病毒（AAV）固有的生物学优势，基因治疗领域在其应用方面取得了重大进展。靶向AAV载体是通过对衣壳进行基因或化学修饰以满足用户特定目的而产生的。然而，这一过程可能导致病毒蛋白序列同质性、化学计量和功能转导载体单元的失衡，从而带来新的挑战。这篇小型综述重点关注了在开发靶向载体方面正在进行的努力，这些努力无意中给临床应用带来了意想不到的障碍，并对未来方向提供了展望。
