Entering the Modern Era of Gene Therapy

被引：337

作者：

Anguela, Xavier M. ^{[1
]}

High, Katherine A. ^{[1
]}

机构：

[1] Spark Therapeut Inc, Philadelphia, PA 19104 USA

来源：

ANNUAL REVIEW OF MEDICINE, VOL 70 | 2019年 / 70卷

关键词：

gene therapy; lentiviral vectors; adeno-associated viral vectors; immunotherapy; T-CELLS; OPEN-LABEL; FACTOR-IX; VECTOR INTEGRATION; LENTIVIRAL VECTOR; PHASE-I; SAFETY; HEMOPHILIA; AAV; TRANSDUCTION;

D O I：

10.1146/annurev-med-012017-043332

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

100103 [病原生物学]; 100218 [急诊医学];

摘要：

Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. Investigators are advancing with cautious optimism that effective, durable, and safe therapies will provide benefit to patients-not only those with single-gene disorders but those with complex acquired diseases as well. While the strategies being translated from the lab to the clinic are numerous, this review focuses on the clinical research that has forged the gene therapy field as it currently stands.

引用

页码：273 / 288

页数：16

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