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治疗性基因组编辑的艰巨经济学。

The Daunting Economics of Therapeutic Genome Editing.

机构信息

Innovative Genomics Institute, University of California, Berkeley, Berkeley, California.

California Institute for Quantitative Biosciences, California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, California.

出版信息

CRISPR J. 2019 Oct;2(5):280-284. doi: 10.1089/crispr.2019.0052.

Abstract

There is no shortage of enthusiasm for the clinical potential of CRISPR-based genome editing: many life-changing cures appear to be just around the corner. However, as mature genetic therapies reach the market, it seems that million-dollar price tags are the new normal. Several factors contribute to the extreme pricing of next-generation medicines, including the need to recoup development costs, the undeniable value of these powerful therapies, and the inherent technical challenges of manufacture and delivery. CRISPR technology has been hailed as a great leveler and a democratizing force in biomedicine. But for this principle to hold true in clinical contexts, therapeutic genome editing must avoid several pitfalls that could substantially limit access to its transformative potential, especially in the developing world.

摘要

人们对基于 CRISPR 的基因组编辑的临床潜力充满热情:许多改变生活的治疗方法似乎即将出现。然而,随着成熟的基因疗法进入市场,百万美元的价格标签似乎已成常态。下一代药物的极端定价有几个因素,包括收回开发成本的需要、这些强大疗法不可否认的价值,以及制造和输送的固有技术挑战。CRISPR 技术被誉为生物医学中的伟大平衡器和民主化力量。但是,为了使这一原则在临床环境中成立,治疗性基因组编辑必须避免可能严重限制其变革潜力的接入的几个陷阱,尤其是在发展中国家。

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