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CRISPR/Cas9,肥胖症潜在治疗中的强大新型基因组编辑工具。

CRISPR/Cas9, the Powerful New Genome-Editing Tool for Putative Therapeutics in Obesity.

机构信息

Laboratorio de Neurociencias Moleculares e Integrativas, Escuela de Medicina División Ciencias de la Salud, Universidad Anáhuac Mayab, A.P. 96 Cordemex C.P, 97310, Mérida, Yucatán, Mexico.

Intercontinental Neuroscience Research Group, Mérida, Yucatán, Mexico.

出版信息

J Mol Neurosci. 2018 May;65(1):10-16. doi: 10.1007/s12031-018-1076-4. Epub 2018 May 7.

Abstract

The molecular technology known as clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) is revolutionizing the field of medical research and deepening our understanding of numerous biological processes. The attraction of CRISPR/Cas9 lies in its ability to efficiently edit DNA or modulate gene expression in living eukaryotic cells and organisms, a technology that was once considered either too expensive or scientifically risky. CRISPR/Cas9 has been successfully applied in agriculture to develop the next generation of disease-resistant plants. Now, the capability of gene editing has been translated to the biomedical area, focusing on the future of medicine faced with drug-resistant microbes by selectively targeting genes involved in antibiotic resistance, for example, or finding the ultimate strategy for cancer or HIV. In this regard, it was recently demonstrated that an injection of cancer-fighting CRISPR-modified white blood cells in a patient suffering from metastatic lung cancer could lead to promising results. Researchers and bioethicists are debating questions about the regulation of CRISPR/Cas9 that must be addressed. While legal challenges surround the use of this technique for genetically modifying cell lines in humans, we review the basic understanding of CRISPR/Cas9 and discuss how this technology could represent a candidate for treatment of non-communicable diseases in nutrition, such as obesity.

摘要

被称为成簇规律间隔短回文重复(CRISPR)/CRISPR 相关蛋白(Cas)的分子技术正在彻底改变医学研究领域,并加深我们对许多生物学过程的理解。CRISPR/Cas9 的吸引力在于其能够有效地编辑真核细胞和生物体中的 DNA 或调节基因表达,这项技术曾经被认为要么过于昂贵,要么在科学上存在风险。CRISPR/Cas9 已成功应用于农业领域,用于开发新一代抗病虫害的植物。现在,基因编辑的能力已经被转化到生物医学领域,专注于解决未来医学所面临的抗药性微生物问题,例如通过选择性地靶向与抗生素耐药性相关的基因,或者寻找治疗癌症或 HIV 的终极策略。在这方面,最近有研究表明,向患有转移性肺癌的患者注射抗癌的 CRISPR 修饰的白细胞,可以带来有希望的结果。研究人员和生物伦理学家正在就必须解决的 CRISPR/Cas9 监管问题进行辩论。虽然围绕着这项技术在人类细胞系中的基因修饰的使用存在法律挑战,但我们回顾了对 CRISPR/Cas9 的基本理解,并讨论了这项技术如何成为治疗营养相关非传染性疾病(如肥胖症)的候选方法。

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