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通过基因疗法共同攻克大疱性表皮松解症。

The joint battle to tackle epidermolysis bullosa through gene therapy.

机构信息

Holostem Terapie Avanzate, s.r.l, Modena, Italy.

Centre for Regenerative Medicine 'Stefano Ferrari', University of Modena and Reggio Emilia, Modena, Italy.

出版信息

Trends Mol Med. 2022 Jul;28(7):533-535. doi: 10.1016/j.molmed.2022.05.001. Epub 2022 May 19.

Abstract

Efforts are dedicated to definitively tackle skin lesions plaguing patients with epidermolysis bullosa (EB), a devastating genetic disorder affecting the integumentary system. Both in vivo gene therapy, as recently reported by Gurevich et al., and combined ex vivo cell and gene therapy strategies are under investigation. Here, we address the advantages and disadvantages of these different approaches.

摘要

研究人员致力于彻底解决困扰大疱性表皮松解症 (EB) 患者的皮肤损伤问题,该病是一种严重的遗传性皮肤系统疾病。目前正在研究体内基因治疗(如 Gurevich 等人最近报道的那样)和体外细胞与基因联合治疗策略。在此,我们将讨论这些不同方法的优缺点。

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