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Alternative routes for parenteral nucleic acid delivery and related hurdles: highlights in RNA delivery 肠外核酸递送的替代途径及相关障碍:RNA 递送方面的亮点
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-19 DOI: 10.1080/17425247.2024.2405207
Joana Cunha, Fátima V Ventura, Christine Charrueau, António José Ribeiro
Nucleic acid-based therapies are promising advancements in medicine. They offer unparalleled efficacy in treating previously untreatable diseases through precise gene manipulation techniques. Howev...
以核酸为基础的疗法是医学领域前景广阔的进步。通过精确的基因操作技术,核酸疗法在治疗以前无法治疗的疾病方面具有无与伦比的疗效。然而...
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引用次数: 0
Sustained intra-cellular siRNA release from Poly(hypenCapswithspaceRetainColl1rginine) multilayered nanoparticles for prolonged gene silencing. 从聚(带空间保留环)多层纳米粒子中持续释放细胞内 siRNA 以延长基因沉默时间
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-18 DOI: 10.1080/17425247.2024.2405206
Alice Ng Jie Ying,Yang Fei Tan,Yee Shan Wong,Subbu Venkatraman
BACKGROUNDSustained siRNA release from nanocarriers is difficult to achieve inside the cell after entry: typically, all nanocarriers exhibit burst release of the cargo into the cytoplasm.RESEARCH DESIGN AND METHODSLayer by layer (LbL) nanoparticles (NPs) can be constructed so that they escape endosomes intact, and subsequently exhibit sustained release of the cargo. Our work quantifies intra-cellular siRNA release from multilayered NPs, evaluates mechanism behind the sustained release, and optimizes the duration of release.RESULTSIntra-cellular studies showed that nanoparticles developed with 4 layers of polyL-arginine, alternated with 3 layers of siRNA layers was able to elicit effective and prolonged SPARC knockdown activity over 21 days with a single dose treatment. For the first time, we have quantified the amounts of released siRNA in the cytoplasm and the amount of siRNA remaining inside the nanoparticles at each timepoint. Furthermore, we have correlated the amount of released siRNA within cells by LbL NPs to the cellular knockdown efficiency of multilayered delivery system.CONCLUSIONSThis methodology may provide an excellent screening tool for assessing the duration of gene silencing by various nanocarrier formulations.
背景纳米载体进入细胞后很难在细胞内实现 siRNA 的持续释放:通常,所有纳米载体都表现出货物向细胞质的猝发释放。结果细胞内研究表明,由 4 层聚精氨酸层和 3 层 siRNA 层交替组成的纳米颗粒能在 21 天的单剂量治疗中有效并持久地敲除 SPARC。我们首次量化了细胞质中释放的 siRNA 量和纳米颗粒中残留的 siRNA 量。此外,我们还将 LbL NPs 在细胞内释放的 siRNA 量与多层递送系统的细胞敲除效率联系起来。
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引用次数: 0
Development of in vitro biopharmaceutics tools for predicting the bioavailability of subcutaneously injected monoclonal antibodies and oligonucleotides 开发用于预测皮下注射单克隆抗体和寡核苷酸生物利用度的体外生物药剂学工具
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-16 DOI: 10.1080/17425247.2024.2403469
Karin Somby, Valerio Campagna, Manuel V. Sanchez-Felix, Ben Forbes, Driton Vllasaliu
Published in Expert Opinion on Drug Delivery (Just accepted, 2024)
发表于《给药专家意见》(刚刚接受,2024 年)
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引用次数: 0
3D printed personalized therapies for pediatric patients affected by adrenal insufficiency 针对肾上腺功能不全儿科患者的 3D 打印个性化疗法
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-13 DOI: 10.1080/17425247.2024.2399706
Lucía Rodríguez-Pombo, Concepción Gallego-Fernández, Anna Kirstine Jørgensen, Carlos Javier Parramon-Teixidó, Carme Cañete-Ramirez, Maria Josep Cabañas-Poy, Abdul W. Basit, Carmen Alvarez-Lorenzo, Alvaro Goyanes
Adrenal insufficiency is usually diagnosed in children who will need lifelong hydrocortisone therapy. However, medicines for pediatrics, in terms of dosage and acceptability, are currently unavaila...
肾上腺功能不全通常在儿童中确诊,他们需要终生接受氢化可的松治疗。然而,儿科用药在剂量和可接受性方面目前尚不完善。
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引用次数: 0
Redefining drug therapy: innovative approaches using catalytic compartments. 重新定义药物疗法:利用催化区的创新方法。
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-11 DOI: 10.1080/17425247.2024.2403476
Yasemin Leyla Mustafa,Arianna Balestri,Xinan Huang,Cornelia Palivan
INTRODUCTIONRapid excretion of drug derivatives often results in short drug half-lives, necessitating frequent administrations. Catalytic compartments, also known as nano- and micro-reactors, offer a solution by providing confined environments for in situ production of therapeutic agents. Inspired by natural compartments, polymer-based catalytic compartments have been developed to improve reaction efficiency and enable site-specific therapeutic applications.AREAS COVEREDPolymer-based compartments provide stability, permeability control, and responsiveness to stimuli, making them ideal for generating localized compounds/signals. These sophisticated systems, engineered to carry active compounds and enable selective molecular release, represent a significant advancement in pharmaceutical research. They mimic cellular functions, creating controlled catalytic environments for bio-relevant processes. This review explores the latest advancements in synthetic catalytic compartments, focusing on design approaches, building blocks, active molecules, and key bio-applications.EXPERT OPINIONCatalytic compartments hold transformative potential in precision medicine by improving therapeutic outcomes through precise, on-site production of therapeutic agents. While promising, challenges like scalable manufacturing, biodegradability, and regulatory hurdles must be addressed to realize their full potential. Addressing these will be crucial for their successful application in healthcare.
引言 药物衍生物的快速排泄往往导致药物半衰期较短,因此需要频繁给药。催化室(也称为纳米和微反应器)提供了一种解决方案,它为原位生产治疗药物提供了封闭的环境。受天然隔室的启发,我们开发了基于聚合物的催化隔室,以提高反应效率,实现特定部位的治疗应用。涵盖领域基于聚合物的隔室具有稳定性、渗透性控制和对刺激的响应性,是产生局部化合物/信号的理想选择。这些复杂的系统可携带活性化合物并实现选择性分子释放,是制药研究的一大进步。它们模仿细胞功能,为生物相关过程创造受控催化环境。本综述探讨了合成催化元件的最新进展,重点关注设计方法、构建模块、活性分子和关键生物应用。专家观点催化元件通过现场精确生产治疗剂来改善治疗效果,在精准医疗领域具有变革潜力。催化元件虽然前景广阔,但要充分发挥其潜力,还必须应对可扩展制造、生物降解性和监管障碍等挑战。解决这些问题对其在医疗保健领域的成功应用至关重要。
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引用次数: 0
Aerosol delivery in interstitial lung diseases - breakthrough or lost cause? 间质性肺病的气溶胶输送--突破还是迷途?
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-04-29 DOI: 10.1080/17425247.2024.2348659
Carsten Ehrhardt
Published in Expert Opinion on Drug Delivery (Ahead of Print, 2024)
发表于《给药专家意见》(2024 年,印刷版前)
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引用次数: 0
Multi-functional nanomedicines for combinational cancer immunotherapy that transform cold tumors to hot tumors 用于组合癌症免疫疗法的多功能纳米药物,将冷肿瘤转化为热肿瘤
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-04-29 DOI: 10.1080/17425247.2024.2348656
Hanhee Cho, Kwangmeyung Kim
Currently, cancer immunotherapy is widely used as a groundbreaking method that can completely cure advanced cancers. However, this new immunotherapy has the challenge of low patient response, which...
目前,癌症免疫疗法作为一种能够彻底治愈晚期癌症的突破性方法被广泛应用。然而,这种新的免疫疗法也面临着病人反应低的挑战,这就需要更多的研究来解决这个问题。
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引用次数: 0
Design of dry powder inhalers to improve patient outcomes: it’s not just about the device 设计干粉吸入器以改善患者疗效:这不仅仅是设备的问题
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-04-17 DOI: 10.1080/17425247.2024.2343894
Jeffry G. Weers
Up to 50% of asthma/COPD patients make critical errors in dose preparation and dose inhalation with current marketed DPIs which negatively impact clinical outcomes. Others fail to adhere to their c...
多达 50% 的哮喘/慢性阻塞性肺病患者在使用目前市场上销售的干粉吸入器进行剂量准备和剂量吸入时会出现严重错误,从而对临床效果产生负面影响。还有一些患者未能坚持按疗程用药...
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引用次数: 0
Doxorubicin conjugates: a practical approach for its cardiotoxicity alleviation 多柔比星共轭物:减轻心脏毒性的实用方法
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-04-16 DOI: 10.1080/17425247.2024.2343882
Abed Alqader Ibrahim, Hamdi Nsairat, Mazen Al-Sulaibi, Mohamed El-Tanani, Areej M. Jaber, Zainab Lafi, Rahmeh Barakat, Duaa Azmi Abuarqoub, Ismail Sami Mahmoud, Sherine O Obare, Alaa A. A. Aljabali, Alaaldin M. Alkilany, Walhan Alshaer
Doxorubicin (DOX) emerges as a cornerstone in the arsenal of potent chemotherapeutic agents. Yet, the clinical deployment of DOX is tarnished by its proclivity to induce severe cardiotoxic effects,...
多柔比星(DOX)是强效化疗药物的基石。然而,DOX 的临床应用却因其易诱发严重的心脏毒性反应而蒙上了一层阴影,...
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引用次数: 0
Expert opinion on antimicrobial therapies: is there enough scientific evidence to state that targeted therapies outperform non-targeted ones? 关于抗菌疗法的专家意见:是否有足够的科学证据表明靶向疗法优于非靶向疗法?
IF 6.6 2区 医学 Q1 PHARMACOLOGY & PHARMACY Pub Date : 2024-04-15 DOI: 10.1080/17425247.2024.2340661
Cristina Yus, Enrique Gámez, Manuel Arruebo
Different active and passive strategies have been developed to fight against pathogenic bacteria. Those actions are undertaken to reduce the bacterial burden while minimizing the possibilities to d...
人们开发了不同的主动和被动策略来对抗致病细菌。采取这些行动的目的是减少细菌的负担,同时最大限度地降低致病菌感染的可能性。
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引用次数: 0
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Expert Opinion on Drug Delivery
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