Application of topical gentamicin-a new era in the treatment of genodermatosis  

作　　者：Shan Wang Zhou Yang Ying Liu Mu-Tong Zhao Juan Zhao Huan Zhang Zong-Yang Liu Xiao-Ling Wang Lin Ma Yong-Hong Yang 

机构地区：[1]Department of Dermatology,Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,No.56 South Li Shi Road,Xi Cheng District,Beijing 100045,China [2]Department of Pharmacy.Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,No.56 South Li Shi Road,Xi Cheng District,Beijing 100045,China [3]China National Clinical Research Center for Respiratory Diseases,Department of Respiratory Medicine,Beijing Children's Hospital,Capital Medical University,National Center for Children’s Health,No.56 South Li Shi Road,Xi Cheng District,Beijing 100045,China

出　　处：《World Journal of Pediatrics》2021年第6期568-575,共8页世界儿科杂志（英文版）

基　　金：supported by Children's Medicine Research Project of Beijing Children's Hospital,Capital Medical University(YZZD202002).

摘　　要：Background The clinical use of gentamicin always lies in its antimicrobial activity in the past as an aminoglycoside antibiotic.However,in the past decade,there were considerable interests in therapeutic approaches in treating hereditary diseases.Some of the genodermatosis is caused by nonsense mutations that create premature termination codons and lead to the production of truncated or non-functional proteins.Gentamicin could induce readthrough of nonsense mutations and enable the synthesis of full-length proteins.We focus on previous publications on topical application of gentamicin and review its utility in genetic skin diseases.Data sources We search the MEDLINE through PubMed,EMBASE databases,and the Clinical Trials Registry Platform from January 1960 to July 2020 using the key search terms"gentamicin,topical gentamicin,genodermatosis,genetic skin diseases".Results The application of gentamicin in genodermatosis yielded promising results,both in vivo and in vitro,including Nagashima-type palmoplantar keratosis,epidermolysis bullosa,Hailey-Hailey disease,hereditary hypotrichosis simplex of the scalp,etc.Conclusions Topical gentamicin is a potential treatment option for genodermatosis caused by nonsense mutation.

关 键 词：Epidermolysis bullosa GENTAMICIN Nagashima-type palmoplantar keratosis Nonsense mutation READTHROUGH 

分 类 号：R751[医药卫生—皮肤病学与性病学]