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相关概念视频

Preclinical Development: Overview01:28

Preclinical Development: Overview

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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Clinical Trials: Overview01:11

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Drug Discovery: Overview01:26

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Drug Administration and Therapy Phases: Overview01:26

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Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
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In Vitro Drug Release Testing: Overview, Development and Validation01:10

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In vitro dissolution and drug release tests assess how quickly and how much of a drug is released from its dosage form into an aqueous medium under standardized laboratory conditions. These tests are essential tools in pharmaceutical development and quality assurance, offering insight into the drug's performance before clinical use.During formulation development, dissolution testing identifies incomplete or inconsistent drug release issues. It also supports decisions on selecting the optimal...
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Drug Regulation01:25

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Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
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相关实验视频

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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
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药物开发 药物开发

Malik White1,2, Rikke Han Kofoed3,4,5, Chinaza Lilian Dibia1,2

  • 1University of Toronto, Toronto, ON, Canada.

Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 26, 2025
PubMed
概括

将静脉内腺相关病毒 (AAV) 载体与聚焦超声波 (FUS) 结合起来,可以增强跨血脑屏障的基因传递. 这种非侵入性方法允许精确或广泛的基因治疗大脑疾病.

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In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
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科学领域:

  • 神经科学是一个神经科学.
  • 基因治疗 基因治疗
  • 医疗成像医学成像

背景情况:

  • 再组合腺相关病毒 (AAV) 对脑部疾病有前途,但在侵入性传递和血脑屏障 (BBB) 透方面面临挑战.
  • 静脉注射 (IV) 输入 静脉注射 (IV) 输入 静脉注射 由于BBB,AAV的输送效率低下,限制了治疗范围.
  • 新的AAV变体和MRI引导的聚焦超声波 (FUS) 为增强大脑传导提供了潜在的解决方案.

研究的目的:

  • 为了研究静脉注射的联合疗效. 透BBB的AAV和FUS用于优化大脑中的基因传递.
  • 为治疗神经退行性疾病实现广泛和特定区域的基因转导.

主要方法:

  • C57BL/6小鼠接受了输液治疗. 在 AAV-PHP.V1.CAG.TdTomato (V1) 和 AAV9.CAG.EYFP.中使用.
  • 聚焦超声波 (FUS) 应用于注射后针对各种大脑区域.
  • 免疫组织化学和共聚焦显微镜用于分娩后3周的分析.

主要成果:

  • 在AAV9和V1载体中,FUS显著增强了针对性的基因传递.
  • V1 载体显示维持了全球大脑表达与FUS增强的局部丰富.
  • 结合方法为精确的 (AAV9) 或广泛的 (V1) 基因疗法提供了一个非侵入性策略.

结论:

  • 这种联合的AAV和FUS策略提高了整个BBB的基因传递效率.
  • 这种方法有可能改善神经退行性疾病 (如阿尔茨海默氏症和帕金森病) 的治疗方法.