Genome Editing in Medicine: Tools and Challenges

Q3 Medicine Acta Medica Lituanica Pub Date : 2021-08-17 DOI:10.15388/Amed.2021.28.2.8
Gunda Petraitytė, E. Preiksaitiene, V. Mikštienė
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引用次数: 1

Abstract

Studies which seek fundamental, thorough knowledge of biological processes, and continuous advancement in natural sciences and biotechnology enable the establishment of molecular strategies and tools to treat disorders caused by genetic mutations. Over the years biological therapy evolved from using stem cells and viral vectors to RNA therapy and testing different genome editing tools as promising gene therapy agents. These genome editing technologies (Zinc finger nucleases, TAL effector nucleases), specifically CRISPR-Cas system, revolutionized the field of genetic engineering and is widely applied to create cell and animal models for various hereditary, infectious human diseases and cancer, to analyze and understand the molecular and cellular base of pathogenesis, to find potential drug/treatment targets, to eliminate pathogenic DNA changes in various medical conditions and to create future “precise medication”. Although different concerning factors, such as precise system delivery to the target cells, efficacy and accuracy of editing process, different approaches of making the DNA changes as well as worrying bioethical issues remain, the importance of genome editing technologies in medicine is undeniable. The future of innovative genome editing approach and strategies to treat diseases is complicated but interesting and exciting at once for all related parties – researchers, clinicians, and patients.
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医学中的基因组编辑:工具和挑战
研究寻求对生物过程的基本、彻底的了解,以及自然科学和生物技术的不断进步,有助于建立治疗基因突变引起的疾病的分子策略和工具。多年来,生物治疗从使用干细胞和病毒载体发展到RNA治疗,并测试不同的基因组编辑工具作为有前景的基因治疗剂。这些基因组编辑技术(锌指核酸酶、TAL效应核酸酶),特别是CRISPR-Cas系统,彻底改变了基因工程领域,并广泛应用于创建各种遗传性、传染性人类疾病和癌症的细胞和动物模型,分析和理解发病机制的分子和细胞基础,寻找潜在的药物/治疗靶点,消除各种医疗条件下致病性DNA的变化,创造未来的“精确药物”。尽管仍存在不同的相关因素,如精确的系统递送到靶细胞、编辑过程的有效性和准确性、改变DNA的不同方法以及令人担忧的生物伦理问题,但基因组编辑技术在医学中的重要性是不可否认的。创新的基因组编辑方法和治疗疾病的策略的未来是复杂的,但对所有相关方——研究人员、临床医生和患者来说,这既有趣又令人兴奋。
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来源期刊
Acta Medica Lituanica
Acta Medica Lituanica Medicine-General Medicine
CiteScore
0.70
自引率
0.00%
发文量
33
审稿时长
16 weeks
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