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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

Clinical Trials: Overview

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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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药物开发 药物开发

Kimberly Scearce-Levie1, Pierce Ogden1, Isabel Albuja1

  • 1Manifold Bio, Boston, MA, USA.

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PubMed
概括
此摘要是机器生成的。

研究人员发现了新的血脑屏障 (BBB) 受体,称为转细胞体,以改善中枢神经系统疾病 (如阿尔茨海默氏症) 的药物输送. 这一发现扩大了治疗大脑疾病的治疗选择,通过增强针对性传递在BBB.

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科学领域:

  • 神经科学是一个神经科学.
  • 药理学 药理学是指药理学的学科.
  • 生物技术是生物技术.

背景情况:

  • 中枢神经系统 (CNS) 疾病带来了治疗挑战,因为血脑屏障 (BBB) 限制了治疗机会.
  • 目前用于穿越BBB的受体介导细胞转移 (RMT) 方法缺乏中枢神经系统特异性和细胞类型选择性.
  • 验证的BBB受体 ("门户") 的有限数量限制了治疗开发.

研究的目的:

  • 对BBB班车和门户进行全面的体内评估.
  • 确定新的门户目标,以提高整个BBB的宏分子运输.
  • 扩大验证的门户的景观,以改善中枢神经系统的药物输送.

主要方法:

  • 在小鼠中对3000多名候选人进行了选,对68个潜在的门户目标进行了选.
  • 采用了新的集成体内查技术,mCodesTM,用于同时进行定量组织分布分析.
  • 对新型门户进行了详细的药理动力学和生物分布分析.

主要成果:

  • 发现了"转细胞体",这是一个扩展的门户目标集,可以在BBB中实现RMT.
  • 与已知的目标 (如TfR和CD98.8) 相比,识别了具有独特属性的新型门户.
  • 发现了新的门户,适合针对细胞外 (如Abeta) 和细胞内 (如Tau) 病理.

结论:

  • 新发现的门户受体为中枢神经系统的输送提供了对生物分布的增强控制.
  • 这些门户网站可以提高细胞类型的特异性,区域定位和减少外围影响.
  • 扩展的Portal景观为开发生物制剂提供了基础,用于精确设计中枢神经系统疾病 (如阿尔茨海默氏症) 的组织分布.