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Updated: May 22, 2025

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皮肤基因疗法的进展:从内外看

Mark J Osborn1, Sidharth Panda2, Theresa M Reineke2

  • 1Medical School, Department of Pediatrics, Division of Blood and Marrow Transplant and Cellular and Gene Therapy, University of Minnesota, Minneapolis, MN 55455, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|March 13, 2025
PubMed
概括

细胞和基因疗法通过克服皮肤的屏障功能,为遗传性皮肤疾病提供了有希望的治疗方法. 这些方法包括局部,皮内和全身传递方法,衰退性缩性表皮溶解 (bullosa) 作为一个关键例子.

关键词:
消化性表皮溶解 牛肉基因编辑 基因编辑基因治疗的基因疗法基因皮肤病的基因皮肤病.树皮细胞 树皮细胞

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