共查询到18条相似文献,搜索用时 109 毫秒
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徐应永 《中国生物工程杂志》2021,40(12):95-103
基因治疗是将外源性遗传物质通过多种方法转移到靶细胞中,用来治疗特定疾病。基因治疗对某些单基因遗传病有一次治愈的可能,或将成为未来治疗人类疾病的重要手段。但基因治疗仍存在诸多挑战,如长期的安全性和疗效数据、可及性以及监管等。从基因治疗的基本概念和历史出发,结合基因治疗产品的开发现状和未来,进行讨论。 相似文献
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惠利健 《中国细胞生物学学报》2019,(4)
<正>在人类同疾病抗争过程中,使用的药物经历了从天然动植物产物、人工合成小分子、生物大分子,到最近以嵌合抗原受体T细胞(CAR-T)为代表的细胞治疗和基因治疗类药物的演变。从长远看,随着细胞制备、基因编辑和递送等技术的进一步完善和成熟,未来10~20年将会是一个细胞治疗、基因治疗等新型疗法争奇 相似文献
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基因治疗是指通过在基因水平上操纵或修饰细胞内基因的表达来治疗疾病的一种生物医学手段。近年来,基因治疗技术逐渐成熟,产业化加速,不断有重磅产品获批,已成为生物医药领域继小分子、大分子之后的一条新热门赛道。本文从基础研究、临床研究和产品获批等方面对2023年基因治疗的态势进行了分析,结果发现治疗及递送新技术的不断涌现及疾病生物学研究的深入,推动基因治疗发展进入快车道,适应证范围不断拓展,临床潜力不断获得验证,产品加速上市。2023年,基因替代治疗、基因编辑治疗和RNA治疗等基因疗法先后迎来多款突破性新产品上市,递送技术的开发和优化取得重要进展,同时基因治疗领域潜在的安全风险和有效性仍需进一步的长期跟踪研究;基因治疗的可及性也有待多渠道来进一步提高。最后,本文也对基因治疗领域的未来发展进行了展望。 相似文献
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<正>基因治疗是将目的基因导入患者的特定组织或细胞进行适当有调控的表达,以纠正或补偿因基因缺陷或异常而引起的疾病,从而达到治疗疾病的目的~([1-2])。基因治疗可以被用于治疗血友病等单基因遗传性疾病以及癌症、神经退行性疾病、眼科疾病和心血管疾病等多基因疾病。2017年,美国FDA批准了一种基因治疗产品Luxturna~(TM),该 相似文献
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引言基因治疗,(GeneTherapy),是指在基因水平对人类遗传疾病进行治疗。具体地说,基因治疗是利用基因转移或基因调控的手段,将正常基因转入疾病患者机体细胞中,取代突变基因,表达所缺乏的基因产物,或者通过关闭或降低 异常表达的基因等途径,达到治疗某些人类疾病的方法。目前的科学发展水平还只能在体细胞水平上对单基因遗传病进行基因治疗,随着科学的发展将会有越来越多的疾病能用此法进行根治。 相似文献
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Gene therapy, recently frequently investigated, is an alternative treatment method that introduces therapeutic genes into a cancer cell or tissue to cause cell death or slow down the growth of the cancer. This treatment has various strategies such as therapeutic gene activation or silencing of unwanted or defective genes; therefore a wide variety of genes and viral or nonviral vectors are being used in studies. Gene therapy strategies in cancer can be classified as inhibition of oncogene activation, activation of tumor suppressor gene, immunotherapy, suicide gene therapy and antiangiogenic gene therapy. In this review, we explain gene therapy, gene therapy strategies in cancer, approved gene medicines for cancer treatment and future of gene therapy in cancer. Today gene therapy has not yet reached the level of replacing conventional therapies. However, with a better understanding of the mechanism of cancer to determine the right treatment and target, in the future gene therapy, used as monotherapy or in combination with another existing treatment options, is likely to be used as a new medical procedure that will make cancer a controllable disease. 相似文献
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Kampen KR 《The Journal of membrane biology》2011,242(2):69-74
Membrane proteins are involved in the prognosis of the most common forms of cancer. Membrane proteins are the hallmark of
a cancer cell. The overexpressed membrane receptors are becoming increasingly important in cancer cell therapy. Current renewing
therapy approaches based on receptor overexpression include; antibody therapy, nanocarrier drug delivery, and fluorescent
tumor imaging in surgery. Gene profiling reveals cancer specific signatures and may identify membrane proteins that are related
to cancer progression and lead to the development of improved therapy strategies in the future. 相似文献
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Seymour LW 《Trends in biotechnology》2006,24(8):347-349
Gene therapy encompasses a spectrum of therapeutic strategies, ranging from the compelling concept of using wild type copies of genes to correct the root cause of recessive genetic disorders through to using genes to mediate powerful and selective toxicity to cancer cells. Inspirational for the general public as well as the bioscience community, gene therapy has been grabbing the headlines--for good and bad reasons--regularly for the past 15 years. In this personal appraisal, Professor Len Seymour assesses the progress of gene therapy in the UK and what it might deliver in the foreseeable future. 相似文献
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Samantha L. Ginn Ian E. Alexander Michael L. Edelstein Mohammad R. Abedi Jo Wixon 《The journal of gene medicine》2013,15(2):65-77
To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical trials from official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our June 2012 update, we have entries on 1843 trials undertaken in 31 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and which genes have been transferred. Details of the analyses presented, and our searchable database are available on The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical . We also provide an overview of the progress being made in clinical trials of gene therapy approaches around the world and discuss the prospects for the future. Copyright © 2013 John Wiley & Sons, Ltd. 相似文献
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《遗传学报》2017,(9)
The adoptive transfer of engineered T cells for the treatment of cancer, autoimmunity, and infectious disease is a rapidly growing field that has shown great promise. Gene editing holds tremendous potential for further improvements of T cell therapy. Here we review the applications of gene editing in various T cell therapies, focusing on antiviral strategies and cancer immunotherapies, and discuss the challenges and future prospects. 相似文献
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《Molecular medicine today》1998,4(11):494-504
Prostate cancer is the most common neoplasm in men and a significant cause of mortality in affected patients. Despite significant advances, current methods of treatment are effective only in the absence of metastatic disease. Gene therapy offers a renewed hope of using the differential characteristics of normal and malignant tissue in constructing treatment strategies. Several clinical trials in prostate cancer gene therapy are currently under way, using immunomodulatory genes, anti-oncogenes, tumor suppressor genes and suicide genes. A continued understanding of the etiological mechanisms involved in the establishment and progression of prostate cancer, along with advances in gene therapy technology, should make gene therapy for prostate cancer therapeutically valuable in the future. 相似文献
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《Microbes and infection / Institut Pasteur》2021,23(8):104829
Gene therapy has become a treatment method for many diseases. Adeno-associated virus (AAV) is one of the most common virus vectors, is also widely used in the gene therapy field. During the past two decades, the retrograde axonal transportability of AAV has been discovered and utilized. Many studies have worked on the retrograde axonal transportability of AAV, and more and more people are interested in this field. This review describes the current application, influence factors, and mechanism of retrograde axonal transportability of AAV and predicted its potential use in disease treatment in near future. 相似文献
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Gene therapy is a promising and rapidly developing field of modern medicine and is expected to improve or even cure the diseases that are incurable with classical therapies. The logics of the development of gene therapy in the nearest future will require the systems wherein a regulation is possible for expression of therapeutic genes. The review considers the currently available regulated gene therapeutic systems, which can be divided into two main classes. One includes the systems wherein external inducers are used to trigger therapeutic gene expression. Systems of the other class are autoregulated and function without an external inducer. The most important first-class expression systems are based on the regulation by tetracycline, rapamycin derivative-induced dimerization, steroid hormones, regulatory RNAs, and physical factors. The most important systems of the second class are regulated by oxygen or glucose levels. 相似文献