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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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...
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Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure01:16

Treatment for Pulmonary Arterial Hypertension: Oxygen Therapy for Respiratory Failure

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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,...
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Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists01:23

Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists

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Prostacyclin receptor agonists are a class of therapeutic agents integral to managing pulmonary arterial hypertension (PAH). These drugs operate by mimicking the action of prostaglandin I2, or PGI2, a naturally occurring compound in the body.
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
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Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists01:18

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

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Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
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Pulmonary Hypertension: Classification and Pathogenesis01:30

Pulmonary Hypertension: Classification and Pathogenesis

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Pulmonary hypertension (PH) is a severe health condition in which the mean pulmonary arterial pressure increases to 25 mmHg or more, even when the body is at rest. This high pressure in the blood vessels that transport blood from the heart to the lungs can cause various symptoms, including shortness of breath, can lead to right heart failure, and significantly affect the overall quality of life.
There are various classifications for PH, each relating to different underlying causes and also...
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COPD: Management Using Bronchodilators and Corticosteroids01:26

COPD: Management Using Bronchodilators and Corticosteroids

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Chronic obstructive pulmonary isease (COPD) involves a group of progressive lung disorders characterized by persistent airflow limitation and chronic respiratory symptoms. Asthma-COPD Overlap Syndrome (ACOS), encompassing features of both asthma and Chronic obstructive pulmonary disease (COPD), is a group of progressive lung disorders that includes chronic bronchitis, emphysema, and refractory (non-reversible) asthma. ACOS leads to complex clinical presentations that combine the inflammatory...
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A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
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Inhaled Gene Transfer for Pulmonary Circulation.

Jaume Aguero1,2, Lahouaria Hadri3, Nadjib Hammoudi3

  • 1Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave Levy Place, #1030, New York, NY, 10029, USA. jaimeaguero30@hotmail.com.

Methods in Molecular Biology (Clifton, N.J.)
|December 3, 2016
PubMed
Summary

Gene therapy offers a novel approach to treat pulmonary hypertension (PH) by targeting vascular remodeling. This study details a method for gene transfer to the distal vasculature in animal models, showing promise for pulmonary vascular diseases.

Keywords:
Adeno-associated virusAirway deliveryGene therapyLarge animal modelPulmonary hypertensionPulmonary vascular diseaseRight ventricular failureVascular remodeling

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Area of Science:

  • Cardiovascular Medicine
  • Pulmonary Medicine
  • Gene Therapy

Background:

  • Chronic pulmonary hypertension (PH) leads to right ventricular failure and high mortality.
  • Current therapies offer limited impact on pulmonary vascular remodeling.
  • Gene therapy presents a novel strategy to address PH limitations.

Purpose of the Study:

  • To describe a procedure for gene transfer to the distal vasculature in animal models of PH.
  • To evaluate the outcomes of this gene therapy intervention.
  • To explore gene therapy as a promising approach for pulmonary vascular diseases.

Main Methods:

  • Development and application of airway delivery of viral vectors.
  • Procedure for achieving vector transduction in the distal vasculature of animal models.
  • Evaluation of intervention outcomes in PH models.

Main Results:

  • Feasibility of gene transfer to the pulmonary vascular system demonstrated in rodent models.
  • Successful vector transduction achieved in the distal vasculature of animal models.
  • The described procedure shows potential for treating pulmonary vascular diseases.

Conclusions:

  • Gene therapy, via airway delivery of viral vectors, is a feasible strategy for PH.
  • This approach allows for selective pulmonary vasculature targeting with minimal systemic effects.
  • The described method offers a promising new avenue for treating pulmonary vascular diseases.