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靶向递药在听力损失基因治疗中的应用。

The applications of targeted delivery for gene therapies in hearing loss.

机构信息

The Biotechnology and Drug Development Research Laboratory, Curtin Medical School & Curtin Health Innovation Research Institute, Curtin University, Perth, Western Australia, Australia.

Hearing Therapeutics Department, Ear Science Institute Australia, Queen Elizabeth II Medical Centre, Perth, Western Australia, Australia.

出版信息

J Drug Target. 2023 Jul;31(6):585-595. doi: 10.1080/1061186X.2023.2216900. Epub 2023 Jun 1.

Abstract

Gene therapies are becoming more abundantly researched for use in a multitude of potential treatments, including for hearing loss. Hearing loss is a condition which impacts an increasing number of the population each year, with significant burdens associated. As such, this review will present the concept that delivering a gene effectively to the inner ear may assist in expanding novel treatment options and improving patient outcomes. Historically, several drawbacks have been associated with the use of gene therapies, some of which may be overcome targeted delivery. Targeted delivery has the potential to alleviate off-target effects and permit a safer delivery profile. Viral vectors have often been described as a delivery method, however, there is an emerging depiction of the potential for nanotechnology to be used. Resulting nanoparticles may also be tuned to allow for targeted delivery. Therefore, this review will focus on hearing loss, gene delivery techniques and inner ear targets, including highlighting promising research. Targeted delivery is a key concept to permitting gene delivery in a safe effective manner, however, further research is required, both in the determination of genes to use in functional hearing recovery and formulating nanoparticles for targeted delivery.

摘要

基因疗法在越来越多的潜在治疗方法中得到了广泛的研究,包括听力损失。听力损失是一种每年影响越来越多人群的疾病,伴随着显著的负担。因此,本综述将提出这样一个概念,即有效地将基因递送到内耳可能有助于扩大新的治疗选择并改善患者的治疗效果。从历史上看,基因疗法的使用存在一些缺陷,其中一些可以通过靶向传递来克服。靶向传递有可能减轻非靶向效应,并允许更安全的传递方式。病毒载体通常被描述为一种传递方法,然而,纳米技术也有可能被用于基因治疗。由此产生的纳米颗粒也可以进行调整以实现靶向传递。因此,本综述将重点关注听力损失、基因传递技术和内耳靶点,包括突出有前途的研究。靶向传递是允许以安全有效的方式进行基因传递的关键概念,然而,需要进一步的研究,包括确定用于功能性听力恢复的基因和为靶向传递而配制纳米颗粒。

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