Zahid Sarwar, Brownell Isaac
Dermatology Service, Memorial Sloan-Kettering Cancer Center.
J Drugs Dermatol. 2008 Apr;7(4):405-8.
Patients with xeroderma pigmentosum (XP) have defective DNA repair and are at a high risk for cutaneous malignancies. Standard treatments for XP are limited in scope and effectiveness. Understanding the molecular etiology of XP has led to the development of novel therapeutic approaches, including enzyme and gene therapies. One new topical treatment utilizing bacteriophage T4 endonuclease 5 (T4N5) in a liposomal lotion is currently in clinical trials and has received a Fast Track designation from the FDA. Gene therapy for XP, while making leaps in preclinical studies, has been slower to develop due to tactical hurdles, but seems to have much potential for future treatment. If these treatments prove effective in lowering the risk of cancer in patients with XP, they may also be found useful in reducing skin cancers in other at-risk patient populations.
着色性干皮病（XP）患者存在DNA修复缺陷，患皮肤恶性肿瘤的风险很高。XP的标准治疗方法在范围和效果上都有限。对XP分子病因的了解促使了新治疗方法的发展，包括酶疗法和基因疗法。一种新的局部治疗方法，即在脂质体乳液中使用噬菌体T4内切酶5（T4N5），目前正在进行临床试验，并已获得美国食品药品监督管理局（FDA）的快速通道指定。XP的基因疗法虽然在临床前研究中取得了进展，但由于策略障碍，其发展较为缓慢，但似乎在未来治疗中具有很大潜力。如果这些治疗方法被证明能有效降低XP患者患癌风险，那么它们也可能对降低其他高危患者群体的皮肤癌发病率有用。
