Therapeutic Potential of CRISPR/Cas in Hashimoto's Thyroiditis: A Comprehensive Review.

IF 3.8 4区 医学 Q2 GENETICS & HEREDITY
Apoorva Upreti, Sayali Mukherjee
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Abstract

Hashimoto's thyroiditis (HT) is a commonly occurring illness of autoimmune endocrine origin. It is usually present in the pediatric age group along with other well-known diseases, such as type 1 insulin-dependent diabetes. The defining feature of this disease is the immune-- mediated attack on the thyroid gland resulting in the destruction of thyroid tissues and cells. Given that HT frequently affects family members, it is well-recognized that individuals are genetically predisposed to this disease. Patients with HT also display a significantly increased risk for several different cancers, justifying the eminent need for the development of therapies for managing and treating HT. Gene editing has made several advancements in the field of molecular biology and has turned out to become a promising approach to correct several autoimmune diseases. Currently, CRISPR/Cas, a nuclease-based editing technique, is publicized as a promising tool for curing several genetic diseases and cancers. However, very limited research has been conducted as of now on autoimmune disease management and cure via CRISPR/Cas technique. This review provides an account of the potential candidate genes associated with Hashimoto's thyroiditis, and only a few animal and human models have been generated via the CRISPR/Cas gene editing technique. Mouse models of autoimmune thyroiditis generated through the CRISPR/Cas gene editing technique by targeting the candidate genes will provide us with a deeper insight into the pathophysiology of HT and further pave the way for the immunomodulation of HT via gene editing.

CRISPR/Cas 在桥本氏甲状腺炎中的治疗潜力:全面回顾。
桥本氏甲状腺炎(HT)是一种常见的自身免疫性内分泌疾病。它通常与其他众所周知的疾病(如1型胰岛素依赖型糖尿病)同时出现在儿童群体中。这种疾病的主要特征是免疫介导的对甲状腺的攻击导致甲状腺组织和细胞的破坏。由于甲状腺肿大经常会影响家庭成员,因此人们普遍认为个人具有患这种疾病的遗传易感性。甲状腺机能亢进症患者罹患多种癌症的风险也明显增加,因此亟需开发用于控制和治疗甲状腺机能亢进症的疗法。基因编辑技术在分子生物学领域取得了多项进展,已成为纠正多种自身免疫性疾病的有效方法。目前,CRISPR/Cas(一种基于核酸酶的编辑技术)被认为是治疗多种遗传疾病和癌症的有效工具。然而,到目前为止,通过 CRISPR/Cas 技术治疗自身免疫性疾病的研究非常有限。本综述介绍了与桥本氏甲状腺炎相关的潜在候选基因,目前只有少数动物和人类模型是通过CRISPR/Cas基因编辑技术生成的。通过CRISPR/Cas基因编辑技术靶向候选基因建立的自身免疫性甲状腺炎小鼠模型将使我们对桥本氏甲状腺炎的病理生理学有更深入的了解,并进一步为通过基因编辑技术对桥本氏甲状腺炎进行免疫调节铺平道路。
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来源期刊
Current gene therapy
Current gene therapy 医学-遗传学
CiteScore
6.70
自引率
2.80%
发文量
46
期刊介绍: Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.
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