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徐应永 《中国生物工程杂志》2020,40(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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H.-R. GREGORIUS 《Biological journal of the Linnean Society. Linnean Society of London》1984,23(2-3):157-165
For a plant selection model with frequency-independent viabilities, fertilities and selfing rates, it is shown that apart from global fixation, for certain parameter combinations a protected polymorphism and facultative fixation (either allele may become fixed according to initial frequencies) may both occur. Facultative fixation requires different selling rates for the dominant and recessive type. Protection of the polymorphism requires resource allocation for male and female function. In this connection the problem of purely genetically caused population extinction is discussed.
For general frequency dependence and regular segregation, the chances for establishment of a completely recessive gene are compared to those of a completely dominant gene. It is proven that the process of establishment of the recessive gene, despite a fitness advantage, may be considerably endangered by drift effects if random mating prevails. The recessive gene may reach the same effectivity in establishment as a dominant gene, only if the recessive homozygote mates exclusively with its own type during the period of establishment. 相似文献
For general frequency dependence and regular segregation, the chances for establishment of a completely recessive gene are compared to those of a completely dominant gene. It is proven that the process of establishment of the recessive gene, despite a fitness advantage, may be considerably endangered by drift effects if random mating prevails. The recessive gene may reach the same effectivity in establishment as a dominant gene, only if the recessive homozygote mates exclusively with its own type during the period of establishment. 相似文献
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In this article, we systematically review Dr. Wu Lien-Teh's academic achievements and outstanding contributions in the prevention and control of the plague epidemic in northeast China and introduce the development of the earliest public health epidemic prevention system in China in order to commemorate the 140th anniversary of Dr. Wu Lien-Teh's birth. We hope that this article will provide insights into the effective prevention and control of emerging infectious diseases as well as the current worldwide pandemic of COVID-19, facilitating the improvement and development of public health systems in China and around the globe. 相似文献
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