基因治疗皮肤脆弱性疾病：新一代。
Gene Therapy for Skin Fragility Diseases: The New Generation.
机构信息
Department of Dermatology, Charles C. Gates Center for Regenerative Medicine, University of Colorado School of Medicine, Anschutz Medical Campus, Aurora, Colorado, USA.
出版信息
J Invest Dermatol. 2019 Aug;139(8):1634-1637. doi: 10.1016/j.jid.2019.04.001.
PMID:31331444
Abstract
Ex vivo gene therapy is a promising approach to treat devastating skin fragility diseases. March et al. and Takashima et al. report that programmable nucleases-TALENs and CRISPR/Cas9-can safely and efficiently correct genetic defects in cultured adult skin cells, paving the way for broader clinical applications of gene therapies in dermatology.
摘要
体外基因治疗是治疗严重皮肤脆弱疾病的一种很有前途的方法。March 等人和 Takashima 等人报告称，可编程核酸酶-TALEN 和 CRISPR/Cas9 可安全有效地纠正培养的成人皮肤细胞中的遗传缺陷，为基因疗法在皮肤病学中的更广泛临床应用铺平了道路。
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