Jove
Visualize
お問い合わせ
JoVE
x logofacebook logolinkedin logoyoutube logo
JoVEについて
概要リーダーシップブログJoVEヘルプセンター
著者向け
出版プロセス編集委員会範囲と方針査読よくある質問投稿
図書館員向け
推薦の声購読アクセスリソース図書館諮問委員会よくある質問
研究
JoVE JournalMethods CollectionsJoVE Encyclopedia of Experimentsアーカイブ
教育
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab Manual教員リソースセンター教員サイト
利用規約
プライバシーポリシー
ポリシー

関連する概念動画

Preclinical Development: Overview01:28

Preclinical Development: Overview

5.7K
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...
5.7K
Clinical Trials: Overview01:11

Clinical Trials: Overview

4.5K
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...
4.5K
Drug Discovery: Overview01:26

Drug Discovery: Overview

10.9K
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...
10.9K
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

1.1K
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...
1.1K
In Vitro Drug Release Testing: Overview, Development and Validation01:10

In Vitro Drug Release Testing: Overview, Development and Validation

279
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...
279
Drug Regulation01:25

Drug Regulation

2.7K
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...
2.7K

こちらも読む

関連記事

共著者、ジャーナル、引用グラフによってこの研究に関連する記事。

並び替え
Same author

Prescriptive Approach to Convective Perfusion of the Bilateral Thalami for Gene Therapy.

Operative neurosurgery (Hagerstown, Md.)·2026
Same author

Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem.

Molecular therapy. Advances·2026
Same author

Clinical GDNF delivery methods for Parkinson's disease.

Journal of Parkinson's disease·2026
Same author

Factors influencing accelerated progression in behavioral variant frontotemporal dementia.

Journal of neurology·2026
Same author

CT-guided robotic placement of multiple convective delivery cannulas.

Journal of neurosurgery·2026
Same author

Flexible Multimodal Neuroimaging Fusion for Alzheimer's Disease Progression Prediction.

Applications of medical artificial intelligence. AMAI (Workshop) (4th : 2024 : Taejon-si, Korea)·2026
Same journal

Evidence for progressive neurodegeneration in iatrogenic cerebral amyloid angiopathy.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
Same journal

Human brain connectome profiles mediate the relationship between pathology burden and clinical phenotypes in Alzheimer's disease.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
Same journal

Kat5 cKO mouse replicates biological domain signatures associated with Alzheimer's disease.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
Same journal

Evaluation of CSF and plasma tau species as fluid surrogate candidates for tau PET in prodromal to moderate Alzheimer's disease.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
Same journal

Associations of self-reported obstructive sleep apnea with cognition and dementia risk in cognitively unimpaired middle-aged adults.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
Same journal

Inflammation profiles in Alzheimer's disease relate to cognition and neurodegeneration.

Alzheimer's & dementia : the journal of the Alzheimer's Association·2026
関連記事をすべて見る

関連する実験動画

Updated: Jan 7, 2026

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
05:10

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

Published on: December 11, 2016

10.1K

薬物開発

Mark H Tuszynski1,2, Douglas W Scharre3, Gabriel C Leger1

  • 1University of California - San Diego, La Jolla, CA, USA.

Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 25, 2025
PubMed
まとめ
この要約は機械生成です。

この第1相臨床試験では、AAV2-BDNF遺伝子治療が軽度アルツハイマー病（AD）および軽度認知障害（MCI）に対して安全であることが示されています。治療を受けた患者は脳代謝の回復を示しており、神経修復治療の可能性を示唆しています。

キーワード:
AAV2-BDNF遺伝子治療アルツハイマー病軽度認知障害脳代謝神経修復

さらに関連する動画

In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
08:04

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

Published on: May 11, 2021

3.3K
Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells
05:45

Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells

Published on: October 10, 2025

451

関連する実験動画

Last Updated: Jan 7, 2026

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
05:10

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

Published on: December 11, 2016

10.1K
In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
08:04

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

Published on: May 11, 2021

3.3K
Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells
05:45

Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells

Published on: October 10, 2025

451

科学分野:

  • 神経科学
  • 遺伝子治療
  • 神経学

背景:

  • 脳由来神経栄養因子（BDNF）は、神経細胞の生存、機能、および認知プロセスに不可欠である。
  • アルツハイマー病（AD）の動物モデルでは、BDNF遺伝子導入が認知パフォーマンスを向上させることが示されている。
  • 以前の研究では、BDNFの有益性が前臨床モデルで実証されており、ヒト試験への進展を支持している。

研究 の 目的:

  • バイオマーカーで支持された軽度ADおよび軽度認知障害（MCI）患者におけるAAV2-BDNF遺伝子送達の安全性と有効性を評価すること。
  • ADおよびMCI患者における認知機能および脳代謝に対するAAV2-BDNF遺伝子治療の影響を評価すること。

主な方法:

  • 海馬傍回にAAV2-BDNFをMRIガイド下で注入する第1相臨床試験。
  • 2年間追跡される12例の患者（軽度AD 6例、MCI 6例）の募集。
  • モニタリングのための連続的な認知テストおよびフルオロデオキシグルコース陽電子放出断層撮影（FDG-PET）スキャン。

主要な成果:

  • 軽度AD患者6例にAAV2-BDNF遺伝子治療を投与し、最長18ヶ月間追跡した。
  • AAV2-BDNF治療に関連する重篤な有害事象は報告されなかった。
  • 少なくとも6ヶ月間治療を受けた3例の患者におけるFDG-PETスキャンでは、アルツハイマー病の典型的な低下を逆転させる、頭頂葉における皮質代謝の増加が示された。

結論:

  • 海馬傍回に投与されたAAV2-BDNF遺伝子治療は、軽度AD患者において安全である。
  • 治療は、FDG-PETスキャンによって証明されるように、標的化された脳領域における代謝活性を回復させた。
  • BDNF遺伝子治療は、神経細胞の喪失を防ぎ機能を高めることにより、ADおよびMCIに対する潜在的な神経修復アプローチを提供する。