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| 1 | Non-coding RNAs:the new central dogma of cancer biology显示文摘The central dogma of molecular biology states that the functions of RNA revolve around protein translation.Until the last decade,most researches were geared towards characterization of RNAs as intermediaries in protein translation,namely,messenger RNAs(mRNAs)as temporary copies of genetic information,ribosomal RNAs(rRNAs)as a main component of ribosome,or translators of codon sequence(t RNAs).The statistical reality,however,is that these processes account for less than 2%of the genome,and insufficiently explain the functionality of 98%of transcribed RNAs.Recent discoveries have unveiled thousands of unique non-coding RNAs(ncRNAs)and shifted the perception of them from being'junk'transcriptional products to'yet to be elucidated'—and potentially monumentally important—RNAs.Most ncRNAs are now known as key regulators in various networks in which they could lead to specific cellular responses and fates.In major cancers,ncRNAs have been identified as both oncogenic drivers and tumor suppressors,indicating a complex regulatory network among these ncRNAs.Herein,we provide a comprehensive review of the various ncRNAs and their functional roles in cancer,and the pre-clinical and clinical development of nc RNA-based therapeutics.A deeper understanding of ncRNAs could facilitate better design of personalized therapeutics. | Phei Er Saw Xiaoding Xu Jianing Chen Er-Wei Song | 2021 | Science China(Life Sciences)2021，64，1： | 21 |
| 2 | Pulmonary delivery of siRNA against acute lung injury/acute respiratory distress syndrome显示文摘The use of small interfering RNAs(si RNAs)has been under investigation for the treatment of several unmet medical needs,including acute lung injury/acute respiratory distress syndrome(ALI/ARDS)wherein si RNA may be implemented to modify the expression of pro-inflammatory cytokines and chemokines at the m RNA level.The properties such as clear anatomy,accessibility,and relatively low enzyme activity make the lung a good target for local si RNA therapy.However,the translation of si RNA is restricted by the inefficient delivery of si RNA therapeutics to the target cells due to the properties of naked si RNA.Thus,this review will focus on the various delivery systems that can be used and the different barriers that need to be surmounted for the development of stable inhalable si RNA formulations for human use before si RNA therapeutics for ALI/ARDS become available in the clinic. | Makhloufi Zoulikha Qingqing Xiao George Frimpong Boafo Marwa A.Sallam Zhongjian Chen Wei He | 2022 | Acta Pharmaceutica Sinica B2022，12，2： | 7 |
| 3 | 基因治疗中的核酸药物及非病毒递送载体的研究进展显示文摘基因治疗是针对基因异常相关疾病的终极治疗技术,各种具有不同机制的核酸药物的出现为基因治疗带来了更多的可能性。但是,由于存在体内稳定性差、难以高效进入靶细胞等问题,核酸药物需要载体的帮助而进入目标细胞并到达特定的胞内位置,因此,开发安全高效的核酸递送系统是基因治疗的基石。与病毒载体相比,非病毒载体具有更高的安全性,但转染效率较低。随着纳米技术的发展,非病毒载体的效率得到了显著的提升,进入临床研究的数量逐渐增多。本文简要介绍基因治疗中的核酸药物及其递送载体,对非病毒核酸药物递送技术的瓶颈及进展做综合评述。 | 刘健 | 2022 | 基础医学与临床2022，42，1： | 5 |
| 4 | Downregulation of EphB2 by RNA interference attenuates glial/fibrotic scar formation and promotes axon growth显示文摘The rapid formation of a glial/fibrotic scar is one of the main factors hampering axon growth after spinal cord injury. The bidirectional Eph B2/ephrin-B2 signaling of the fibroblast-astrocyte contact-dependent interaction is a trigger for glial/fibrotic scar formation. In the present study, a new in vitro model was produced by coculture of fibroblasts and astrocytes wounded by scratching to mimic glial/fibrotic scar-like structures using an improved slide system. After treatment with RNAi to downregulate Eph B2, changes in glial/fibrotic scar formation and the growth of VSC4.1 motoneuron axons were examined. Following RNAi treatment, fibroblasts and astrocytes dispersed without forming a glial/fibrotic scar-like structure. Furthermore, the expression levels of neurocan, NG2 and collagen I in the coculture were reduced, and the growth of VSC4.1 motoneuron axons was enhanced. These findings suggest that suppression of Eph B2 expression by RNAi attenuates the formation of a glial/fibrotic scar and promotes axon growth. This study was approved by the Laboratory Animal Ethics Committee of Jiangsu Province, China(approval No. 2019-0506-002) on May 6, 2019. | Jian Wu Zhen-Yu Zhu Zhi-Wei Fan Ying Chen Ri-Yun Yang Yi Li | 2022 | Neural Regeneration Research2022，17，2： | 4 |
| 5 | A 10-miRNA risk score-based prediction model for pathological complete response to neoadjuvant chemotherapy in hormone receptor-positive breast cancer显示文摘Patients with hormone receptor(HR)-positive tumors breast cancer usually experience a relatively low pathological complete response(p CR)to neoadjuvant chemotherapy(NAC).Here,we derived a 10-micro RNA risk score(10-mi RNA RS)-based model with better performance in the prediction of p CR and validated its relation with the disease-free survival(DFS)in 755 HRpositive breast cancer patients(273,265,and 217 in the training,internal,and external validation sets,respectively).This model,presented as a nomogram,included four parameters:the 10-mi RNA RS found in our previous study,progesterone receptor(PR),human epidermal growth factor receptor 2(HER2)status,and volume transfer constant(K).Favorable calibration and discrimination of 10-mi RNA RS-based model with areas under the curve(AUC)of 0.865,0.811,and 0.804 were shown in the training,internal,and external validation sets,respectively.Patients who have higher nomogram score(>92.2)with NAC treatment would have longer DFS(hazard ratio=0.57;95%CI:0.39–0.83;P=0.004).In summary,our data showed the 10-mi RNA RS-based model could precisely identify more patients who can attain p CR to NAC,which may help clinicians formulate the personalized initial treatment strategy and consequently achieves better clinical prognosis for patients with HRpositive breast cancer. | Chang Gong Ziliang Cheng Yaping Yang Jun Shen Yingying Zhu Li Ling Wanyi Lin Zhigang Yu Zhihua Li Weige Tan Chushan Zheng Wenbo Zheng Jiajie Zhong Xiang Zhang Yunjie Zeng Qiang Liu RStephanie Huang Andrzej LKomorowski Eddy SYang François Bertucci Francesco Ricci Armando Orlandi Gianluca Franceschini Kazuaki Takabe Suzanne Klimberg Naohiro Ishii Angela Toss Mona PTan Mathew A Cherian Erwei Song | 2022 | Science China(Life Sciences)2022，65，11： | 3 |
| 6 | Applications and developments of gene therapy drug delivery systems for genetic diseases显示文摘Genetic diseases seriously threaten human health and have always been one of the refractory conditions facing humanity.Currently,gene therapy drugs such as siRNA,shRNA,antisense oligonucleotide,CRISPR/Cas9 system,plasmid DNA and miRNA have shown great potential in biomedical applications.To avoid the degradation of gene therapy drugs in the body and effectively deliver them to target tissues,cells and organelles,the development of excellent drug delivery vehicles is of utmost importance.Viral vectors are the most widely used delivery vehicles for gene therapy in vivo and in vitro due to their high transfection efficiency and stable transgene expression.With the development of nanotechnology,novel nanocarriers are gradually replacing viral vectors,emerging superior performance.This review mainly illuminates the current widely used gene therapy drugs,summarizes the viral vectors and non-viral vectors that deliver gene therapy drugs,and sums up the application of gene therapy to treat genetic diseases.Additionally,the challenges and opportunities of the field are discussed from the perspective of developing an effective nano-delivery system. | Xiuhua Pan Hanitrarimalala Veroniaina Nan Su Kang Sha Fenglin Jiang Zhenghong Wu Xiaole Qi | 2021 | Asian Journal of Pharmaceutical Sciences2021，16，6： | 3 |
| 7 | 阳离子脂质体在基因治疗中的研究进展显示文摘基因治疗从基因水平上对疾病进行干预已备受关注,阳离子脂质体作为一种非病毒载体,因其制备简单,可降解性等特点,可作为基因治疗的有效载体。该文从结构、制备方法、作用机制及应用等方面阐述了阳离子脂质体在基因治疗中的研究进展。 | 汪琼卉 刘哲鹏 李北雪 | 2022 | 生物医学工程学进展2022，43，2： | 2 |
| 8 | mRNA治疗及其应用前景显示文摘信使核糖核酸(mRNA)是由DNA的一条链作为模板转录而来的携带遗传信息、指导蛋白质合成的一类单链核糖核酸.随着mRNA体外合成中修饰调控/序列优化系统的日益完善和体内递送系统的逐渐成熟,mRNA缺乏稳定性及无法有效递送等缺点已经逐渐被克服,基于mRNA的治疗方法已逐步成为研究热点.通过体外转录技术合成的mRNA,借助脂质纳米颗粒递送系统运送到特定的组织细胞内,由细胞自身的翻译系统翻译出目标蛋白.这些蛋白或是作为抗原激发免疫反应,或是补充细胞内缺少的蛋白以行使功能,最终达到治疗的目的.mRNA治疗具有的制备快速、成本低、安全等优点让它在众多治疗方法中脱颖而出,被广泛地应用于癌症疫苗、感染性疾病疫苗、蛋白替代治疗和罕见病治疗等领域.为了解全球mRNA治疗的开发和研究现状,本文重点对mRNA治疗的修饰调控/序列优化系统、递送系统以及临床研究现状进行分析和总结. | 杭辰悦 徐山杉 吴其峰 张普昱 张宇 徐颖洁 | 2021 | 科学通报2021，66，28： | 2 |
| 9 | 装载siRNA的纳米阳离子脂质体在小鼠体内的药代动力学研究显示文摘目的:探讨siRNA药物的体内药代动力学评价方法,并分析脂质体作为药物载体的优势。方法:采用前期已构建的siRNA表达质粒及装载了该质粒的纳米隐形阳离子脂质体,将实验分为裸siRNA质粒组和siRNA质粒脂质体组,分别进行DNaseⅠ酶切和血清中稳定性的检测。再将20μg siRNA裸质粒及siRNA质粒脂质体通过尾静脉注射进入小鼠体内,分别在注射后0 min、5 min、15 min、30 min、1 h、2 h、4 h、12 h、24 h取血,采用实时荧光定量PCR方法(qPCR)对小鼠体内裸质粒及质粒脂质体进行定量检测,计算各时间点siRNA质粒的浓度。结果:裸质粒在DNaseⅠ中仅20 min就全部降解,质粒脂质体降解较缓慢,到24 h仍有(20.16±2.47)%的DNA残留;裸质粒在80%血清中20 min时仅残留(11.03±0.92)%,在1 h时基本全部降解,质粒脂质体在80%血清中24 h时仍残留(10.26±1.04)%,与裸质粒组相比,脂质体作为载体使siRNA表达质粒在DNaseⅠ及血清中的稳定性显著提高(P<0.05)。在小鼠体内脂质体包裹的质粒半衰期约为2 h,而裸质粒仅为6 min,差异具有统计学意义(P<0.05)。结论:利用qPCR方法可以完成siRNA脂质体的体内检测。阳离子脂质体能够保护核酸类药物进入体内,是一种高效的递送载体,同时脂质体能够提高药物的体内稳定性,延长药物的作用时间。 | 问天娇 陈欣然 白靖 郑颖 王雅鹃 于佩佩 崔京霞 | 2022 | 癌变.畸变.突变2022，34，6： | 1 |
| 10 | 差异表达的非编码RNA在脓毒症诱导的急性呼吸窘迫综合征中的作用显示文摘急性呼吸窘迫综合征(acute respiratory distress syndrome,ARDS)是一种ICU中常见的病死率较高的临床综合征,ARDS发生时,富含蛋白质的液体在肺泡中积聚,阻止肺部充满足够的空气,从而导致到达血液中的氧气减少,发生低氧血症^([1-2])。急性肺损伤(acute lung injury,ALI)由严重的感染、外伤、休克、吸入有害气体及中毒等直接或间接因素引起的全身炎症反应综合征在肺部的表现,以肺泡及肺实质发生急性炎症为主要病理特征,其特点是发生低氧血症、非心源性肺水肿、肺顺应性降低和广泛的毛细血管渗漏^([3])。 | 闵思敏 张小楠 丁渡山 刘赛赛 李言 | 2022 | 临床肺科杂志2022，27，10： | 1 |
| 11 | 小核酸药物非临床特点和药理毒理评价策略显示文摘通过与靶向信使RNA结合,小核酸药物能特异性地沉默疾病基因来治疗疾病。目前,已有12种小核酸药物在欧美国家上市,还有更多药物处于各个研发阶段。小核酸药物虽然来自人工合成,但因其自身特点不能单纯按照化学药物进行非临床评价。本文结合已上市药物的研究资料和相关指导原则,分别从成药性、药效学、药动学及安全性评价四方面对小核酸药物非临床特点和评价策略进行阐述。 | 王恒 李华 汪溪洁 邱云良 汤纳平 黄芳华 常艳 | 2022 | 中国新药杂志2022，31，12： | 1 |
| 12 | 基因编辑技术在现代医学中的应用与挑战显示文摘基因编辑技术已经成为现代医学领域中引人注目的工具,其中RNA干扰(RNA interference,RNAi)和CRISPR-Cas9系统是近年来发展最迅速的基因编辑技术。RNAi通过介导特定RNA分子的降解或抑制来实现基因沉默,而CRISPR-Cas9系统可以剪切和编辑特定的DNA序列。RNAi和CRISPR-Cas9系统已经在多个领域展示出了巨大的潜力,同时它们在应用中也面临着一些挑战。本文介绍RNAi和CRISPR-Cas9系统的作用机制,简述了两种基因编辑技术在不同领域的应用,并对其面临的挑战和应用前景进行展望。 | 李香灵 崔安芳 黄延红 马晓磊 | 2023 | 生物化工2023，9，4： | 1 |
| 13 | CRISPR-CasRx knock-in mice for RNA degradation显示文摘The RNA editing tool CRISPR-CasRx has provided a platform for a range of transcriptome analysis tools and therapeutic approaches with its broad efficacy and high specificity.To enable the application of CasRx in vivo,we established a Credependent CasRx knock-in mouse.Using these mice,we specifically knocked down the expression of Meis1 and Hoxb13 in cardiomyocytes,which induced cardiac regeneration after myocardial infarction.We also knocked down the lnc RNA Mhrt in cardiomyocytes with the CasRx knock-in mice,causing hypertrophic cardiomyopathy.In summary,we generated a Credependent CasRx knock-in mouse that can efficiently knock down coding gene and lnc RNA expression in specific somatic cells.This in vivo CRISPR-CasRx system is promising for gene function research and disease modeling. | Jiacheng Li Dekun Zhu Shengshou Hu Yu Nie | 2022 | Science China(Life Sciences)2022，65，11： | 1 |
| 14 | Nanoparticles in the diagnosis and treatment of vascular aging and related diseases显示文摘Aging-induced alternations of vasculature structures,phenotypes,and functions are key in the occurrence and development of vascular aging-related diseases.Multiple molecular and cellular events,such as oxidative stress,mitochondrial dysfunction,vascular inflammation,cellular senescence,and epigenetic alterations are highly associated with vascular aging physiopathology.Advances in nanoparticles and nanotechnology,which can realize sensitive diagnostic modalities,efficient medical treatment,and better prognosis as well as less adverse effects on non-target tissues,provide an amazing window in the field of vascular aging and related diseases.Throughout this review,we presented current knowledge on classification of nanoparticles and the relationship between vascular aging and related diseases.Importantly,we comprehensively summarized the potential of nanoparticles-based diagnostic and therapeutic techniques in vascular aging and related diseases,including cardiovascular diseases,cerebrovascular diseases,as well as chronic kidney diseases,and discussed the advantages and limitations of their clinical applications. | Hui Xu Shuang Li You-Shuo Liu | 2022 | Signal Transduction and Targeted Therapy2022，7，8： | 1 |
| 15 | 蚓激酶同工酶降解乙型肝炎抗原并保护肝功能显示文摘目的蚓激酶同工酶(LKIs)作为肠溶胶囊的有效成分,用于治疗血栓性疾病已有30多年历史。近年来,LKIs在其他危重疾病中的研究时有报道。本文关注LKIs在乙型肝炎方面的作用。方法乙型肝炎表面抗原(HBs Ag)、核心抗原(HBcAg)和e抗原(HBeAg)分别与不同浓度LKIs孵育,观察这些蛋白质的降解和估计肽链的切割位点。Hep G2.2.15细胞与LKIs孵育,采用酶联免疫吸附测定(ELISA)和蛋白质印迹(Western blotting)检测细胞分泌的HBsAg和HbeAg。LKIs灌胃Balb/c小鼠30天,采用ELISA和Western blotting检测其血清HBsAg和HBeAg,免疫组化染色检测肝组织中的HBcAg。采用苏木精-伊红染色分析乙肝病毒转基因小鼠肝组织的损害,并通过ELISA定量分析血清谷草转氨酶(GOT)和谷丙转氨酶(GPT)。腹腔注射后,取大鼠血清和肝组织,测定其中的LKIs含量,从而观察LKIs的吸收。采用LKIs给龙岩麻鸭灌胃30天,通过PCR检测其血清HBV DNA。结果蚓激酶肠溶胶囊的有效成分是含有6种LKIs的复方蛋白酶药物,可以降解HBV编码的蛋白质。LKIs降解HBsAg的位点为K141/P142及R160/F161;HBc Ag为R142/E143;HBeAg为R122/E123。LKIs可显著抑制Hep G2.2.15细胞分泌HBsAg和HBeAg。LKIs灌胃,HBV转基因小鼠血清HBsAg和HBeAg水平及肝组织的HBcAg水平均降低,提示病毒的组装和释放可能受到了抑制。在LKIs处理的转基因小鼠中,血清GPT和GOT水平降低,肝组织溶解数量减少,表明LKIs对小鼠肝细胞具有保护作用。LKIs灌胃龙岩麻鸭,血清中DHBV DNA水平明显下降,停药后出现反弹。结论LKIs通过降解HBs、HBc和HBe蛋白,可能干扰HBV的装配和释放,减少病毒在肝细胞之间的传递,从而对肝细胞起到保护作用。 | 周园 牟丽娴 范士超 王秀梅 曹潇 王学清 张奉学 赵静 魏艳 赫荣乔 | 2022 | 生物化学与生物物理进展2022，49，1： | 0 |
| 16 | 壳聚糖衍生物作为siRNA纳米递送系统材料的研究进展显示文摘小干扰RNA(small interfering RNA,siRNA)在治疗疾病方面具有高效性、特异性、瞬时性等优良特性,是目前药物研发关注的焦点。但裸露的siRNA稳定性差、细胞摄取率低,难以发挥其基因沉默作用,因此需要一种优质的递送系统将其递送至靶细胞中,使其发挥作用。大部分纳米递送系统因毒性、载药问题不利于siRNA的转运,而壳聚糖衍生物具有较高的水溶性、安全性、稳定性,已受到广泛关注。本文综述了壳聚糖衍生物应用于siRNA纳米递送系统的研究进展,为正在进行纳米递送系统研究的科研工作者提供参考。 | 熊峰 苏峰 曹俊如 储晓琴 何广卫 | 2023 | 中国现代应用药学2023，40，13： | 0 |
| 17 | 经气道siRNA递送系统:急性肺损伤治疗的有效策略显示文摘急性肺损伤(acute lung injury,ALI)是一种致死率高的严重呼吸系统疾病,常表现为肺水肿、肺泡通透性增加、炎性细胞聚集和弥漫性肺泡损伤,其最终可导致急性呼吸窘迫综合征(acute respiratory distress syndrome,ARDS)[1-2]。过度的炎症反应是ALI进展的重要危险因素,浸润到肺组织的巨噬细胞和中性粒细胞释放大量细胞因子,诱导过度炎症反应,对肺泡上皮和内皮细胞的产生快速和严重损伤[3]。迄今为止,尽管对ALI/ARDS进行了广泛的研究,然而并没有开发出有效的药物治疗这一疾病,目前针对ALI/ARDS的最佳治疗仍然是保护性通气,其病死率在大约40%左右[4]。因此急需开发创新的ALI/ARDS治疗策略来降低其病死率。基因治疗因其精确化、个体化优势已成为目前非常有前景的潜在疾病治疗手段,通过沉默ALI发病机制中关键基因靶标,阻止甚至逆转疾病发展,为ALI/ARDS开辟了潜在的有效治疗策略。 | 李国瑞 付庭吕 李宁 耿庆 | 2023 | 中华急诊医学杂志2023，32，8： | 0 |
| 18 | 自组装多肽用于药物递送的研究进展显示文摘近年来,自组装多肽(SAP)因具有良好的生物相容性、灵敏的响应性及特定的生物功能而备受关注。SAP在特定情况下能形成规则有序的纳米结构,表现出不同于单分子的优势和特性,已广泛应用于细胞培养、组织工程和药物递送等领域。该文主要对多肽自组装形成的纳米类型及其在药物递送方面的应用进行综述,包括化学小分子及核酸、多肽、蛋白质等生物大分子,总结了SAP作为药物递送载体所面临的挑战,并展望其发展前景。 | 赵特 邹鹏飞 李佳欣 高春生 李志平 | 2023 | 军事医学2023，47，10： | 0 |
| 19 | A hidden translatome in tumors——the coding lncRNAs显示文摘Long noncoding RNAs(lncRNAs)have been extensively identified in eukaryotic genomes and have been shown to play critical roles in the development of multiple cancers.Through the application and development of ribosome analysis and sequencing technologies,advanced studies have discovered the translation of lncRNAs.Although lncRNAs were originally defined as noncoding RNAs,many lncRNAs actually contain small open reading frames that are translated into peptides.This opens a broad area for the functional investigation of lncRNAs.Here,we introduce prospective methods and databases for screening lncRNAs with functional polypeptides.We also summarize the specific lncRNA-encoded proteins and their molecular mechanisms that promote or inhibit cancerous.Importantly,the role of lncRNA-encoded peptides/proteins holds promise in cancer research,but some potential challenges remain unresolved.This review includes reports on lncRNA-encoded peptides or proteins in cancer,aiming to provide theoretical basis and related references to facilitate the discovery of more functional peptides encoded by lncRNA,and to further develop new anti-cancer therapeutic targets as well as clinical biomarkers of diagnosis and prognosis. | Jinsong Wang Wenna Wang Fei Ma Haili Qian | 2023 | Science China(Life Sciences)2023，66，12： | 0 |
| 20 | 载siRNA的PLGA-b-PEG纳米粒制备及初步体外评价显示文摘目的 探讨载小干扰核糖核酸(siRNA)的聚乳酸-羟基乙酸共聚物-b-聚乙二醇(PLGA-b-PEG)纳米粒的制备工艺,并对其进行表征及初步体外评价。方法 采用复乳法制备含药纳米粒。通过透射电子显微镜、原子力显微镜观察形态,Zeta电位/粒度分析仪测定Zeta电位及其粒径,计算包封率,考察稳定性、体外释药,并进行细胞增殖检测。结果 制备的纳米粒呈球形,粒径均匀,平均粒径为(101.5±6.3)nm,Zeta电位为-(31.7±4.5)mV,包封率为(57.6±4.8)%;在4℃条件下放置30 d,粒径和Zeta电位分别为(103.3±5.7)nm和-(32.5±5.2)mV;纳米粒体外可持续释药48 h以上,且超声可加速药物释放;siRNA纳米粒具有较强的肿瘤细胞活性抑制作用。结论 制备的纳米粒理化特性良好,性质稳定,具有一定的超声刺激释药特性。 | 纪刚剑 林雯 史琼枝 李银科 | 2022 | 中国药业2022，31，17： | 0 |
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