Prenatal Gene Therapy for Metabolic Disorders.
Prenatal Gene Therapy for Metabolic Disorders.
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DOI：
10.1097/grf.0000000000000662
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发表时间：
2021-12-01
影响因子：
1.5
通讯作者：
Peranteau WH
中科院分区：
文献类型：
--
作者：
Coons B;Peranteau WH
传统上，基因治疗涉及将外源遗传物质递送至细胞，最常见的是替换导致单基因疾病的缺陷基因。这使得细胞能够产生足量的蛋白质，而这些蛋白质在其他情况下是不存在的，非常适合治疗目的。自 40 多年前诞生以来，基因治疗领域已经显着扩展，现在包括靶向基因编辑策略，包括但不限于成簇规则间隔回文重复序列 (CRISPR)/CRISPR 相关蛋白 9 (Cas9)、转录激活子样效应核酸酶 (TALEN) 和锌指核酸酶 (ZFN)。
Gene therapy has traditionally involved the delivery of exogenous genetic material to a cell—most commonly to replace defective genes causing monogenic disorders. This allows cells to produce proteins that are otherwise absent in sufficient quantities, ideally for a therapeutic purpose. Since its inception over 40 years ago, the field of gene therapy has significantly expanded and now includes targeted gene editing strategies, including, but not limited to, clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), transcription activator–like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs).
影响因子：
5.1
作者：
Endo M;Zoltick PW;Radu A;Jiang Q;Matsui C;Marinkovich PM;McGrath J;Tamai K;Uitto J;Flake AW
通讯作者：
Flake AW