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Toward Combined Cell and Gene Therapy for Genodermatoses.
Cold Spring Harbor Perspectives in Biology ( IF 7.2 ) Pub Date : 2020-05-01 , DOI: 10.1101/cshperspect.a035667
Laura De Rosa 1 , Maria Carmela Latella 1 , Alessia Secone Seconetti 1 , Cecilia Cattelani 2 , Johann W Bauer 3 , Sergio Bondanza 1 , Michele De Luca 2
Affiliation  

To date, more than 200 monogenic, often devastating, skin diseases have been described. Because of unmet medical needs, development of long-lasting and curative therapies has been consistently attempted, with the aim of correcting the underlying molecular defect. In this review, we will specifically address the few combined cell and gene therapy strategies that made it to the clinics. Based on these studies, what can be envisioned for the future is a patient-oriented strategy, built on the specific features of the individual in need. Most likely, a combination of different strategies, approaches, and advanced therapies will be required to reach the finish line at the end of the long and winding road hampering the achievement of definitive treatments for genodermatoses.

中文翻译:

迈向针对遗传性皮肤病的细胞和基因联合治疗。

迄今为止,已经描述了 200 多种单基因、通常是毁灭性的皮肤病。由于未满足的医疗需求,一直在尝试开发长效和治愈性疗法,目的是纠正潜在的分子缺陷。在这篇综述中,我们将专门讨论一些用于临床的联合细胞和基因治疗策略。基于这些研究,未来可以设想的是一种以患者为导向的策略,建立在有需要的个体的具体特征之上。最有可能的是,需要结合不同的策略、方法和先进的疗法才能在漫长而曲折的道路尽头到达终点线,这阻碍了对遗传皮肤病的最终治疗的实现。
更新日期:2020-05-01
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