Ex vivo gene modification therapy for genetic skin diseases-recent advances in gene modification technologies and delivery.
Ex vivo gene modification therapy for genetic skin diseases-recent advances in gene modification technologies and delivery.
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DOI：
10.1111/exd.14314
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发表时间：
2021-07
影响因子：
3.6
通讯作者：
Di WL
中科院分区：
文献类型：
--
作者：
Jayarajan V;Kounatidou E;Qasim W;Di WL
遗传性皮肤病，也称为遗传性皮肤病，是影响皮肤的遗传性疾病，构成了一大群异质性疾病。虽然遗传性皮肤病很少见，患病率低于5万至20万分之一，但它们经常发生在出生时或生命早期，通常是慢性的、严重的，并可能危及生命。由于疾病的负面心理社会影响、身体表现以及缺乏或丧失自主权，患者及其家属的生活质量受到严重损害。目前，还没有治愈这些疾病的方法。体外基因修饰疗法是指在体外对患者细胞中的突变基因进行修饰或校正，然后移植回患者体内，以恢复基因的功能性表达。本文综述了遗传性皮肤病的体外基因修饰治疗策略，重点介绍了在患者细胞中基因修饰和校正以及将基因修饰细胞传递给患者方面的最新进展，并讨论了在该领域进行的基因治疗试验。
Genetic skin diseases, also known as genodermatoses, are inherited disorders affecting skin and constitute a large and heterogeneous group of diseases. While genodermatoses are rare with the prevalence rate of less than 1 in 50,000 – 200,000, they frequently occur at birth or early in life and are generally chronic, severe, and could be life‐threatening. The quality of life of patients and their families are severely compromised by the negative psychosocial impact of disease, physical manifestations, and the lack or loss of autonomy. Currently, there are no curative treatments for these conditions. Ex vivo gene modification therapy that involves modification or correction of mutant genes in patients’ cells in vitro and then transplanted back to patients to restore functional gene expression has being developed for genodermatoses. In this review, the ex vivo gene modification therapy strategies for genodermatoses are reviewed, focusing on current advances in gene modification and correction in patients’ cells and delivery of genetically modified cells to patients with discussions on gene therapy trials which have been performed in this area.
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