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Gene Therapy
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DOI：10.1056/NEJMra1706910.png)
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Abstract
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.
Keywords:
SEVERE COMBINED IMMUNODEFICIENCY
LONG-TERM SAFETY
FACTOR-IX
RETROVIRAL VECTORS
CANINE MODEL
OPEN-LABEL
CELL
HEMOPHILIA
EFFICACY
ACTIVATION
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78.5
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2.7W
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39.7W