Gene Therapy

Katherine A. High

doi:10.1056/NEJMra1706910

2019-08-01

374

PRE

Katherine A. High *

Maria Grazia Roncarolo

DOI：10.1056/NEJMra1706910

原文链接

摘要

En 中文

Those who have followed the gene-therapy field over the decades may be weary of forward-looking positive statements. However, over the past 3 years, six gene-therapy products have been approved for clinical use. This article describes challenges, risks, and advances in gene-therapy clinical research.

Keyword:

SEVERE COMBINED IMMUNODEFICIENCY

LONG-TERM SAFETY

FACTOR-IX

RETROVIRAL VECTORS

CANINE MODEL

OPEN-LABEL

CELL

HEMOPHILIA

EFFICACY

ACTIVATION

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期刊

New England Journal of Medicine

IF:

78.5

论文数:

2.7W

被引数:

44.4W

机构

Stanford University

学者数:

5.8K

论文数: 1.9K

被引数: 529

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