Center for Nanomedicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Department of Ophthalmology, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Departments of Chemical and Biomolecular Engineering and Materials Science and Engineering, Johns Hopkins University, Baltimore, MD, USA.
Center for Nanomedicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Department of Ophthalmology, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Glaucoma Center of Excellence, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Adv Drug Deliv Rev. 2023 Sep;200:115005. doi: 10.1016/j.addr.2023.115005. Epub 2023 Jul 6.
Vision impairment and loss due to posterior segment ocular disorders, including age-related macular degeneration and diabetic retinopathy, are a rapidly growing cause of disability globally. Current treatments consist primarily of intravitreal injections aimed at preventing disease progression and characterized by high cost and repeated clinic visits. Nanotechnology provides a promising platform for drug delivery to the eye, with potential to overcome anatomical and physiological barriers to provide safe, effective, and sustained treatment modalities. However, there are few nanomedicines approved for posterior segment disorders, and fewer that target specific cells or that are compatible with systemic administration. Targeting cell types that mediate these disorders via systemic administration may unlock transformative opportunities for nanomedicine and significantly improve patient access, acceptability, and outcomes. We highlight the development of hydroxyl polyamidoamine dendrimer-based therapeutics that demonstrate ligand-free cell targeting via systemic administration and are under clinical investigation for treatment of wet age-related macular degeneration.
由于后段眼部疾病导致的视力障碍和丧失，包括年龄相关性黄斑变性和糖尿病性视网膜病变，是全球范围内导致残疾的一个快速增长的原因。目前的治疗方法主要是通过玻璃体内注射来防止疾病进展，其特点是成本高且需要多次到诊所就诊。纳米技术为眼部药物输送提供了一个有前途的平台，有可能克服解剖和生理屏障，提供安全、有效和持续的治疗方式。然而，只有少数纳米药物被批准用于后段疾病，而且针对特定细胞的纳米药物或与全身给药兼容的纳米药物更少。通过全身给药针对介导这些疾病的细胞类型，可能为纳米医学带来变革性的机会，并显著改善患者的可及性、可接受性和结果。我们重点介绍了基于羟聚酰胺胺树枝状大分子的治疗药物的开发，这些药物通过全身给药展示了无配体的细胞靶向作用，并正在进行临床试验，以治疗湿性年龄相关性黄斑变性。
