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

Gene Therapy00:59

Gene Therapy

25.7K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.7K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

221
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
221
Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure01:16

Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure

311
Oxygen therapy has emerged as a significant tool in enhancing the quality of life for patients suffering from pulmonary arterial hypertension (PAH). While this therapy has principally been studied on patients with significant hypoxemia, this therapeutic approach helps prevent potential organ damage and can be administered in the comfort of one's home.
Oxygen therapy is vital in increasing and maintaining blood oxygen levels in PAH patients. As a result, it aids in reducing fatigue,...
311
Acute Respiratory Failure-V01:29

Acute Respiratory Failure-V

188
The treatment for acute respiratory failure varies based on factors like the underlying cause, overall health, and severity. A collaborative healthcare team is essential for early detection, often through arterial blood gas analysis. Identifying the cause is the primary goal, with treatment strategies adjusted for ventilation/perfusion (V/Q) mismatch, shunting, or diffusion impairment.
Ensure that patients are monitored continuously for their response to therapy, including changes in...
188
Common Respiratory Disorders01:31

Common Respiratory Disorders

840
Respiratory disorders, a prevalent health concern globally, are generally divided into two primary categories: upper and lower respiratory tract disorders. The categorization is based on the area of the respiratory system they affect.
Upper respiratory disorders impact the airways above the vocal cords, encompassing areas like the nose, sinuses, and throat. Various conditions fall under this category, including the common cold and allergic rhinitis. These disorders can stem from several causes,...
840
Respiratory Capacities01:24

Respiratory Capacities

877
Respiratory capacities are crucial indicators of lung function, representing the maximum amount of air an individual's respiratory system can handle during various breathing phases.
One key metric is the Inspiratory Capacity (IC), which represents the maximum amount of air that can be inhaled with full effort. IC is calculated by summing the tidal volume and inspiratory reserve volume, typically ranging from 2.4 to 3.6 liters.
The Functional Residual Capacity (FRC) represents the air in the...
877

You might also read

Related Articles

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

Sort by
Same author

Perspective of the European Cystic Fibrosis Society on Improving Global Cystic Fibrosis Care.

Pediatric pulmonology·2026
Same author

Postural Influence on Ventilation Efficiency and Relationship With Oxygen-Enhanced MRI in Cystic Fibrosis.

Pediatric pulmonology·2026
Same author

Maternal, Infant, Reproductive and Child Health in Cystic Fibrosis (MATRIARCH_CF): a prospective, observational study to evaluate pregnancy and parenthood in females with cystic fibrosis and health of the offspring in the CFTR-modulator era.

BMJ open respiratory research·2026
Same author

Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.

Gene therapy·2026
Same author

Using the nose as a factory to secrete proteins into the lungs or circulation.

Molecular therapy. Advances·2026
Same author

Function and Structure Relationships With Inflammation Differ in Two Chronic Suppurative Lung Diseases.

Pediatric pulmonology·2026
Same journal

Genome Editing for Familial Hemophagocytic Lymphohistiocytosis: Design Principles, Challenges, and Translational Perspectives.

Human gene therapy·2026
Same journal

Lilly, Ascidian Launch Up-to-$1.9B RNA Exon Editor Collaboration.

Human gene therapy·2026
Same journal

Hyperthermia-Enhanced Adeno-Associated Virus Gene Therapy: Mechanisms, Delivery Technologies, and Translational Potential.

Human gene therapy·2026
Same journal

Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings.

Human gene therapy·2026
Same journal

In Remembrance of Professor Zelig Eshhar: <i>A Life Committed to CAR-T</i>.

Human gene therapy·2026
Same journal

<i>In Vivo</i> Genome Editing Approach to Disrupt Hydroxyacid Oxidase 1 for the Treatment of Primary Hyperoxaluria Type 1.

Human gene therapy·2026
See all related articles

Related Experiment Video

Updated: Aug 29, 2025

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
10:12

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

Published on: October 31, 2012

16.6K

Progress in Respiratory Gene Therapy.

Gerry McLachlan1,2, Eric W F W Alton2,3, A Christopher Boyd2,4

  • 1The Roslin Institute &amp; R(D)SVS, University of Edinburgh, Edinburgh, United Kingdom.

Human Gene Therapy
|September 8, 2022
PubMed
Summary
This summary is machine-generated.

Gene therapy for respiratory diseases shows promise, but challenges remain. Researchers are developing advanced vectors and RNA-based therapies to overcome lung barriers and improve treatment efficacy for inherited and acquired conditions.

Keywords:
AAVgene editinggene therapy vectorslentiviruslung diseaserespiratory

More Related Videos

Protein Transfection of Mouse Lung
04:21

Protein Transfection of Mouse Lung

Published on: May 15, 2013

9.4K
Generation, Amplification, and Titration of Recombinant Respiratory Syncytial Viruses
11:48

Generation, Amplification, and Titration of Recombinant Respiratory Syncytial Viruses

Published on: April 4, 2019

16.0K

Related Experiment Videos

Last Updated: Aug 29, 2025

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
10:12

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

Published on: October 31, 2012

16.6K
Protein Transfection of Mouse Lung
04:21

Protein Transfection of Mouse Lung

Published on: May 15, 2013

9.4K
Generation, Amplification, and Titration of Recombinant Respiratory Syncytial Viruses
11:48

Generation, Amplification, and Titration of Recombinant Respiratory Syncytial Viruses

Published on: April 4, 2019

16.0K

Area of Science:

  • Pulmonary Medicine
  • Gene Therapy
  • Molecular Biology

Background:

  • Gene therapy research for respiratory diseases began in the 1980s, with cystic fibrosis as an early focus.
  • Despite numerous clinical trials, no gene therapy products are currently approved for lung conditions.
  • Initial vector systems demonstrated proof-of-concept but suffered from low efficacy.

Purpose of the Study:

  • To review the progress and challenges in developing gene therapy for respiratory diseases.
  • To discuss various vector systems and therapeutic cargoes for lung delivery.
  • To highlight advancements in gene therapy for specific respiratory indications.

Main Methods:

  • Evaluation of viral and non-viral vector systems for lung gene delivery.
  • Analysis of different therapeutic cargoes, including DNA, mRNA, siRNA, and peptide nucleic acids.
  • Review of strategies to overcome lung-specific delivery barriers and improve transgene expression.

Main Results:

  • Significant advancements in vector technology have been made to enhance efficacy and overcome lung barriers.
  • Newer approaches include RNA-based therapies and gene editing tools, moving beyond simple gene addition.
  • Optimized vectors and therapeutic strategies show improved expression characteristics for transgenes.

Conclusions:

  • Gene therapy for respiratory diseases has evolved significantly, with improved vector systems and diverse therapeutic approaches.
  • Overcoming lung-specific challenges remains critical for successful clinical translation.
  • Continued research into novel vectors and therapeutic modalities holds promise for treating inherited and acquired lung diseases.