Tian Bo, Bilsbury Evan, Doherty Sean, Teebagy Sean, Wood Emma, Su Wenqi, Gao Guangping, Lin Haijiang
Department of Ophthalmology and Visual Sciences, University of Massachusetts Chan Medical School, Worcester, MA 01655, USA.
Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.
Pharmaceutics. 2022 Sep 13;14(9):1931. doi: 10.3390/pharmaceutics14091931.
Ocular drug delivery has been significantly advanced for not only pharmaceutical compounds, such as steroids, nonsteroidal anti-inflammatory drugs, immune modulators, antibiotics, and so forth, but also for the rapidly progressed gene therapy products. For conventional non-gene therapy drugs, appropriate surgical approaches and releasing systems are the main deliberation to achieve adequate treatment outcomes, whereas the scope of "drug delivery" for gene therapy drugs further expands to transgene construct optimization, vector selection, and vector engineering. The eye is the particularly well-suited organ as the gene therapy target, owing to multiple advantages. In this review, we will delve into three main aspects of ocular drug delivery for both conventional drugs and adeno-associated virus (AAV)-based gene therapy products: (1) the development of AAV vector systems for ocular gene therapy, (2) the innovative carriers of medication, and (3) administration routes progression.
眼部药物递送不仅在甾体类药物、非甾体类抗炎药、免疫调节剂、抗生素等药物化合物方面取得了显著进展,而且在快速发展的基因治疗产品方面也有很大进步。对于传统的非基因治疗药物,合适的手术方法和释放系统是实现充分治疗效果的主要考虑因素,而基因治疗药物的“药物递送”范围进一步扩展到转基因构建体优化、载体选择和载体工程。由于多种优势,眼睛是特别适合作为基因治疗靶点的器官。在这篇综述中,我们将深入探讨传统药物和基于腺相关病毒(AAV)的基因治疗产品在眼部药物递送的三个主要方面:(1)用于眼部基因治疗的AAV载体系统的开发,(2)药物的创新载体,以及(3)给药途径的进展。