Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

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

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

The modulation of the blood-brain barrier by focused ultrasound stimulates oligodendrogenesis.

Acta neuropathologica communications·2026
Same author

Parametric optimization of transcranial focused ultrasound neuromodulation of the periaqueductal grey for blood pressure reduction.

Brain stimulation·2026
Same author

Focused ultrasound in pediatric neuro-oncology: Current applications and future directions.

Neuro-oncology advances·2026
Same author

Anti-focus driving signals to mitigate bone heating during focused ultrasound ablation of uterine fibroids with phased arrays.

Physics in medicine and biology·2026
Same author

Focused Ultrasound Treatment of Vitreous Hemorrhage in an In Vivo Rabbit Model.

Ophthalmology science·2026
Same author

Optimization of bacterial colonization in the gut of axenic and conventional zebrafish larvae using live food.

Microbiology spectrum·2026

Related Experiment Video

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

Drug Development.

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
Summary

Combining intravenous adeno-associated virus (AAV) vectors with focused ultrasound (FUS) enhances gene delivery across the blood-brain barrier. This non-invasive method allows for precise or widespread gene therapy for brain disorders.

More Related Videos

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

Related Experiment Videos

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

Area of Science:

  • Neuroscience
  • Gene Therapy
  • Medical Imaging

Background:

  • Recombinant adeno-associated viruses (AAVs) are promising for brain disorders but face challenges with invasive delivery and blood-brain barrier (BBB) penetration.
  • Intravenous (i.v.) AAV delivery is inefficient due to the BBB, limiting therapeutic reach.
  • New AAV variants and MRI-guided focused ultrasound (FUS) offer potential solutions for enhanced brain transduction.

Purpose of the Study:

  • To investigate the combined efficacy of i.v. BBB-penetrating AAVs and FUS for optimizing gene delivery in the brain.
  • To achieve both widespread and region-specific gene transduction for treating neurodegenerative diseases.

Main Methods:

  • C57BL/6 mice received i.v. AAV-PHP.V1.CAG.TdTomato (V1) and AAV9.CAG.EYFP.
  • Focused ultrasound (FUS) was applied to target various brain regions post-injection.
  • Immunohistochemistry and confocal microscopy were used for analysis 3 weeks after delivery.

Main Results:

  • FUS significantly enhanced targeted gene delivery for both AAV9 and V1 vectors.
  • V1 vectors demonstrated maintained global brain expression alongside FUS-enhanced local enrichment.
  • The combined approach offers a non-invasive strategy for precise (AAV9) or widespread (V1) gene therapy.

Conclusions:

  • This combined AAV and FUS strategy improves gene delivery efficiency across the BBB.
  • The approach holds potential for enhanced treatments of neurodegenerative disorders like Alzheimer's and Parkinson's disease.