投资者展望:基因治疗加速发展;在十字路口。

SchimmerJoshua, BreazzanoSteven
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引用次数: 3

摘要

基因治疗领域继续加速发展,最近在许多不同的治疗适应症上取得了成功,这凸显了该平台的潜力。随着该领域不断取得进展,越来越多的长期安全性和有效性数据集将继续确定基因治疗的作用,最终确定它在罕见、严重疾病之外的应用范围。随着患者接触的不断积累,新技术往往会出现意想不到的曲折,基因治疗可能也不例外。也就是说,由于许多疾病除了基因治疗之外没有其他治疗选择,而且发病率和死亡率都很高,因此如果这些疾病出现,该领域似乎可以承受一些小的甚至是大的颠簸。
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Investor Outlook: Gene Therapy Picking up Steam; At a Crossroads.
The gene therapy field continues to pick up steam with recent successes in a number of different therapeutic indications that highlight the potential for the platform. As the field continues to make progress, a growing data set of long-term safety and efficacy data will continue to define gene therapy's role, determining ultimately how widely it may be used beyond rare, serious diseases with high unmet needs. New technologies often take unanticipated twists and turns as patient exposure accumulates, and gene therapy may be no exception. That said, with many diseases that have no other treatment options beyond gene therapy and that present considerable morbidity and mortality, the field appears poised to withstand some minor and even major bumps in the road should they emerge.
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来源期刊
Human Gene Therapy Clinical Development
Human Gene Therapy Clinical Development CRITICAL CARE MEDICINEMEDICINE, RESEARCH &-MEDICINE, RESEARCH & EXPERIMENTAL
CiteScore
7.20
自引率
0.00%
发文量
0
期刊介绍: Human Gene Therapy (HGT) is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes important advances in DNA, RNA, cell and immune therapies, validating the latest advances in research and new technologies.
期刊最新文献
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