Gene Therapy for Epidermolysis Bullosa

被引：60

作者：

Marinkovich, M. Peter ^{[1
,2
]}

Tang, Jean Y. ^{[1
]}

机构：

[1] Stanford Univ, Dept Dermatol, Program Epithelial Biol, Sch Med, Stanford, CA 94305 USA

[2] Palo Alto Vet Affairs Med Ctr, Dept Dermatol, Palo Alto, CA USA

来源：

JOURNAL OF INVESTIGATIVE DERMATOLOGY | 2019年 / 139卷 / 06期

基金：

美国国家卫生研究院;

关键词：

DERMAL-EPIDERMAL JUNCTION; VII COLLAGEN; STEM-CELLS; BONE-MARROW; SKIN; LAMININ-5; INJECTION; TRANSPLANTATION; CORRECTS; IDENTIFICATION;

D O I：

10.1016/j.jid.2018.11.036

中图分类号：

R75 [皮肤病学与性病学];

学科分类号：

100206 ;

摘要：

Epidermolysis bullosa is a family of diseases characterized by blistering and fragility of the skin in response to mechanical trauma. Advances in our understanding of epidermolysis bullosa pathophysiology have provided the necessary foundation for the first clinical trials of gene therapy for junctional and dystrophic epidermolysis bullosa. These therapies show that gene therapy is both safe and effective, with the potential to correct the molecular and clinical phenotype of patients with epidermolysis bullosa. Improvements in gene delivery and in preventing immune reactions will be among the challenges that lie ahead during further therapeutic development.

引用

页码：1221 / 1226

页数：6

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