纳米医学
遗传增强
纳米技术
基因传递
专家意见
医学
计算机科学
风险分析(工程)
生物
重症监护医学
纳米颗粒
基因
材料科学
生物化学
作者
Karen Peynshaert,Joke Devoldere,Stefaan De Smedt,Katrien Remaut
标识
DOI:10.1080/17425247.2023.2167979
摘要
Introduction Retinal disease affects millions of people worldwide, generating a massive social and economic burden. Current clinical trials for retinal diseases are dominated by gene augmentation therapies delivered with recombinant viruses as key players. As an alternative, nanoparticles hold great promise for the delivery of nucleic acid therapeutics as well. Nevertheless, despite numerous attempts, ‘nano’ is in practice not as successful as aspired and major breakthroughs in retinal gene therapy applying nanomaterials are yet to be seen.Areas covered In this review, we summarize the advantages of nanomaterials and give an overview of nanoparticles designed for retinal nucleic acid delivery up to now. We furthermore critically reflect on the predominant issues that currently limit nano to progress to the clinic, where faulty study design and the absence of representative models play key roles.Expert opinion Since the current approach of in vitro – in vivo experimentation is highly inefficient and creates misinformation, we advocate for a more prominent role for ex vivo testing early on in nanoparticle research. In addition, we elaborate on several concepts, including systematic studies and open science, which could aid in pushing the field of nanomedicine beyond the preclinical stage.
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