Making gene editing accessible in resource limited environments: recommendations to guide a first-time user.

IF 4.9 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Frontiers in genome editing Pub Date : 2024-09-25 eCollection Date: 2024-01-01 DOI:10.3389/fgeed.2024.1464531
Shivani Goolab, Janine Scholefield
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Abstract

The designer nuclease, CRISPR-Cas9 system has advanced the field of genome engineering owing to its programmability and ease of use. The application of these molecular scissors for genome engineering earned the developing researchers the Nobel prize in Chemistry in the year 2020. At present, the potential of this technology to improve global challenges continues to grow exponentially. CRISPR-Cas9 shows promise in the recent advances made in the Global North such as the FDA-approved gene therapy for the treatment of sickle cell anaemia and β-thalassemia and the gene editing of porcine kidney for xenotransplantation into humans affected by end-stage kidney failure. Limited resources, low government investment with an allocation of 1% of gross domestic production to research and development including a shortage of skilled professionals and lack of knowledge may preclude the use of this revolutionary technology in the Global South where the countries involved have reduced science and technology budgets. Focusing on the practical application of genome engineering, successful genetic manipulation is not easily accomplishable and is influenced by the chromatin landscape of the target locus, guide RNA selection, the experimental design including the profiling of the gene edited cells, which impacts the overall outcome achieved. Our assessment primarily delves into economical approaches of performing efficient genome engineering to support the first-time user restricted by limited resources with the aim of democratizing the use of the technology across low- and middle-income countries. Here we provide a comprehensive overview on existing experimental techniques, the significance for target locus analysis and current pitfalls such as the underrepresentation of global genetic diversity. Several perspectives of genome engineering approaches are outlined, which can be adopted in a resource limited setting to enable a higher success rate of genome editing-based innovations in low- and middle-income countries.

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在资源有限的环境中实现基因编辑:指导首次使用者的建议。
由于其可编程性和易用性,CRISPR-Cas9 系统这种设计型核酸酶推动了基因组工程领域的发展。这些分子剪刀在基因组工程中的应用为研发人员赢得了 2020 年的诺贝尔化学奖。目前,这项技术在改善全球挑战方面的潜力仍在成倍增长。CRISPR-Cas9 最近在全球北方地区取得的进展显示了其前景,如美国食品及药物管理局批准的用于治疗镰状细胞性贫血和β-地中海贫血症的基因疗法,以及用于异种移植到受终末期肾衰竭影响的人体内的猪肾基因编辑。资源有限、政府对研发的投资仅占国内生产总值的 1%,包括缺乏专业技术人员和知识,这些因素都可能阻碍这一革命性技术在全球南方国家的应用,因为这些国家的科技预算减少。就基因组工程的实际应用而言,成功的基因操作并非易事,它受到目标基因座染色质景观、指导 RNA 选择、实验设计(包括基因编辑细胞的分析)的影响,从而影响到所取得的总体成果。我们的评估主要深入研究高效基因组工程的经济方法,以支持受限于有限资源的首次使用者,目的是在低收入和中等收入国家普及该技术的使用。在此,我们全面概述了现有的实验技术、目标基因座分析的意义以及目前存在的缺陷,如全球遗传多样性代表性不足。我们还概述了基因组工程方法的几个方面,这些方法可以在资源有限的情况下采用,从而提高中低收入国家基于基因组编辑的创新的成功率。
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来源期刊
CiteScore
7.00
自引率
0.00%
发文量
0
审稿时长
13 weeks
期刊最新文献
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