Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa.

Ulrich Koller, Johann W Bauer
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Abstract

Background: Epidermolysis bullosa (EB) is a clinically-heterogeneous genodermatosis with severe manifestations in the skin and other organs. The significant burden this condition places on patients justifies the development of gene therapeutic strategies targeting the genetic cause of the disease.

Methods: Emerging RNA and DNA editing tools have shown remarkable advances in efficiency and safety. Applicable both in ex vivo- and in vivo settings, these gene therapeutics based on gene replacement or editing are either at the pre-clinical or clinical stage.

Results: The recent landmark FDA approvals for gene editing based on CRISPR/Cas9, along with the first FDA-approved redosable in vivo gene replacement therapy for EB, will invigorate ongoing research efforts, increasing the likelihood of achieving local cure via CRISPR-based technologies in the near future.

Conclusions: This review discusses the status quo of current gene therapeutics that act at the level of RNA or DNA, all with the common aim of improving the quality of life for EB patients.

治疗大疱性表皮松解症的新兴 DNA 和 RNA 编辑策略。
背景:大疱性表皮松解症(EB)是一种临床异质性遗传性皮肤病,在皮肤和其他器官有严重的表现。这种疾病给患者带来的沉重负担证明了针对该病遗传病因的基因治疗策略的发展是合理的:方法:新兴的 RNA 和 DNA 编辑工具在效率和安全性方面取得了显著进步。这些基于基因置换或编辑的基因疗法适用于体内外环境,目前处于临床前或临床阶段:最近,美国食品与药物管理局批准了基于CRISPR/Cas9的基因编辑技术,以及美国食品与药物管理局批准的首个用于EB的可重复使用的体内基因置换疗法,这两项具有里程碑意义的举措将为当前的研究工作注入新的活力,在不久的将来,通过基于CRISPR的技术实现局部治愈的可能性将大大增加:本综述讨论了目前在 RNA 或 DNA 水平上发挥作用的基因疗法的现状,所有这些疗法的共同目标都是改善 EB 患者的生活质量。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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