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首页> 外文期刊>Clinics in dermatology >Progress and prospects of skin gene therapy: a ten year history.
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Progress and prospects of skin gene therapy: a ten year history.

机译:皮肤基因治疗的进展和前景:已有十年历史。

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摘要

Significant progress has been made in corrective gene therapy of the skin in the last decade. This includes advances in vector technology, targeted gene expression, gene replacement, gene correction, and the availability of appropriate animal models for a variety of candidate diseases. While non-viral integration of large genes such as essential basement membrane proteins has been mastered, new challenges such as the control of immune responses lie ahead of the research community until skin gene therapy will become clinical reality. Among the first skin diseases patients with junctional epidermolysis bullosa and xeroderma pigmentosum have entered clinical trials.
机译:在过去的十年中,在皮肤的纠正基因治疗方面已经取得了重大进展。这包括载体技术,靶向基因表达,基因替代,基因校正以及各种候选疾病的合适动物模型的可用性方面的进步。尽管已经掌握了诸如必需基底膜蛋白之类的大基因的非病毒整合,但诸如皮肤免疫治疗等新挑战仍摆在研究界面前,直到皮肤基因治疗成为临床现实。在最初的皮肤病患者中,大疱性交界性表皮松解症和色素干性皮肤病患者已进入临床试验。

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