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The dawn of a cure for sickle cell disease through CRISPR‐based treatment: A critical test of equity in public health genomics
Annals of Human Genetics ( IF 1.9 ) Pub Date : 2024-03-22 , DOI: 10.1111/ahg.12558
Gerald Mboowa 1, 2 , Ivan Sserwadda 1 , Stephen Kanyerezi 1 , Stephen Tukwasibwe 3, 4 , Benson Kidenya 5
Affiliation  

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.

中文翻译:

通过基于 CRISPR 的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

公平地获取基因组技术、资源和产品仍然是一个巨大的挑战。这在 2019 年冠状病毒病 (COVID-19) 大流行期间尤其明显,尽管这些疫苗发展迅速,但大多数中低收入国家在首次推出 COVID-19 疫苗期间无法实现至少 10% 的人口疫苗接种覆盖率。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 SCD 负担最重的地区,每年至少有 240,000 名出生的儿童患有这种疾病。 SCD 已从一种可治疗的疾病发展为一种可治愈的疾病。最近,英国医疗监管机构批准了基于成簇规则间隔短回文重复 (CRISPR) 的治疗方法,而美国食品和药物管理局也同样批准了两种 SCD 基因疗法。这是展示公共卫生基因组学公平性的绝佳机会。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并可获得这些治疗方法,并能够拯救数百万人的生命。
更新日期:2024-03-22
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