通过 CRISPR 治疗治愈镰状细胞病的曙光:公共卫生基因组学公平性的关键考验。

IF 1 4区 生物学 Q4 GENETICS & HEREDITY
Gerald Mboowa, Ivan Sserwadda, Stephen Kanyerezi, Stephen Tukwasibwe, Benson Kidenya
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引用次数: 0

摘要

公平获取基因组技术、资源和产品仍然是一项巨大挑战。这一点在 2019 年冠状病毒病(COVID-19)大流行期间尤为明显,当时,尽管 COVID-19 疫苗发展迅速,但大多数中低收入国家在最初的 COVID-19 疫苗推广期间仍无法实现至少 10%的人口接种覆盖率。镰状细胞病(SCD)是一种遗传性单基因红细胞疾病,会影响血红蛋白(一种在体内携带氧气的蛋白质)。在全球范围内,非洲大陆是镰状细胞病发病率最高的地区,每年至少有 24 万名患儿出生。SCD 已经从一种可治疗的疾病发展成为一种可治愈的疾病。最近,英国医疗监管机构批准了通过基于成簇规律性间隔短回文重复(CRISPR)的治疗方法治愈该病,而美国食品药品管理局也同样批准了两种 SCD 基因疗法。这为展示公共卫生基因组学的公平性提供了一个难得的机会。这种基于 CRISPR 的治疗方法价格昂贵,因此需要采取雄心勃勃的行动,以确保在最需要的地方能够负担得起和获得这种治疗方法,并能拯救数百万人的生命。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The dawn of a cure for sickle cell disease through CRISPR-based treatment: A critical test of equity in public health genomics.

Equity in access to genomic technologies, resources, and products remains a great challenge. This was evident especially during the coronavirus disease 2019 (COVID-19) pandemic when the majority of lower middle-income countries were unable to achieve at least 10% population vaccination coverage during initial COVID-19 vaccine rollouts, despite the rapid development of those vaccines. Sickle cell disease (SCD) is an inherited monogenic red blood cell disorder that affects hemoglobin, the protein that carries oxygen through the body. Globally, the African continent carries the highest burden of SCD with at least 240,000 children born each year with the disease. SCD has evolved from a treatable to a curable disease. Recently, the UK medical regulator approved its cure through clustered regularly interspaced short palindromic repeat (CRISPR)-based treatment, whereas the US Food and Drug Administration has equally approved two SCD gene therapies. This presents a remarkable opportunity to demonstrate equity in public health genomics. This CRISPR-based treatment is expensive and therefore, a need for an ambitious action to ensure that they are affordable and accessible where they are needed most and stand to save millions of lives.

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来源期刊
Annals of Human Genetics
Annals of Human Genetics 生物-遗传学
CiteScore
4.20
自引率
0.00%
发文量
34
审稿时长
3 months
期刊介绍: Annals of Human Genetics publishes material directly concerned with human genetics or the application of scientific principles and techniques to any aspect of human inheritance. Papers that describe work on other species that may be relevant to human genetics will also be considered. Mathematical models should include examples of application to data where possible. Authors are welcome to submit Supporting Information, such as data sets or additional figures or tables, that will not be published in the print edition of the journal, but which will be viewable via the online edition and stored on the website.
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