Gene therapies for pulmonary hypertension—from experimental trials to bedside aspects

Meng, Lingling; Liu, Chenggui

doi:10.1016/j.ejcts.2009.06.045

Cited by 3 publications

(1 citation statement)

References 191 publications

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“…Recently, several studies by independent groups have shown the potential therapeutic benefit of modulating a variety of molecular targets using gene therapy [7, 8]. For instance, endothelial NOS, prostacyclin synthase or BMPR2 have been successfully modulated in the pulmonary vasculature leading to improved hemodynamics in animal models of PH [9–11]. …”

Section: Introductionmentioning

confidence: 99%

Inhaled Gene Transfer for Pulmonary Circulation

Agüero

Hadri

Hammoudi

et al. 2016

Methods in Molecular Biology

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Chronic pulmonary hypertension (PH) is associated with right ventricular failure and high mortality regardless of the underlying disease. Currently, therapies can improve clinical outcomes in specific subsets of patients, but have little impact on the progression of pulmonary vascular remodeling. Upon new advances in vector development and delivery techniques, gene therapy is a novel strategy in this field with the potential of overcoming the main limitations of approved drug therapies: modulation of novel anti-remodeling targets and selective pulmonary vasculature targeting with minimal systemic effects. In the recent years, several reports have shown that gene transfer to the pulmonary vascular system is feasible in rodent models of PH. Our group has focused on the translation of airway delivery of viral vectors in small and large animals. Here, we describe a procedure to achieve vector transduction at the distal vasculature in animal models of PH and the methods to evaluate the outcomes of this intervention as a promising new approach in pulmonary vascular diseases.

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Section: Introductionmentioning

confidence: 99%

Inhaled Gene Transfer for Pulmonary Circulation

Agüero

Hadri

Hammoudi

et al. 2016

Methods in Molecular Biology

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Endobronchial Gene Delivery for Pulmonary Hypertension in a Large Animal Model

Bikou¹,

Ishikawa²

2022

Methods in Molecular Biology

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Metabolic gene therapy in a canine with pulmonary hypertension secondary to degenerative mitral valve disease

Katz,

Ohad,

Putter

et al. 2024

Front. Vet. Sci.

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Myxomatous mitral valve disease (MMVD) stands out as the most prevalent acquired canine heart disease. Its occurrence can reach up to 40% in small breed dogs and escalates in geriatric canine populations. MMVD leads to thickening and incomplete coaptation of valve leaflets during systole, resulting in secondary mitral valve regurgitation. Serious complications may arise concurrently with the worsening of mitral valve regurgitation, including left-and right-sided congestive heart failure, and pulmonary hypertension (PH). Ultimately, the PH progression might contribute to the patient’s demise or to the owner’s decision of euthanasia. Most currently available FDA-approved therapies for PH are costly and aim to address the imbalance between vasoconstriction and vasodilation to restore endothelial cell function. However, none of these medications impact the molecular dysfunction of cells or impede the advancement of pulmonary vascular and right ventricular remodeling. Recent evidence has showcased successful gene therapy approaches in laboratory animal models of PH. In this manuscript, we summarize the latest advancements in gene therapy for the treatment of PH in animals. The manuscript incorporates original data showcasing sample presentations, along with non-invasive hemodynamic assessments. Our findings demonstrate that the use of metabolic gene therapy, combining synthetic adeno-associated virus with acid ceramidase, has the potential to significantly reduce the need for drug treatment and improve spontaneously occurring PH in dogs.

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Gene therapies for pulmonary hypertension—from experimental trials to bedside aspects

Cited by 3 publications

References 191 publications

Inhaled Gene Transfer for Pulmonary Circulation

Inhaled Gene Transfer for Pulmonary Circulation

Endobronchial Gene Delivery for Pulmonary Hypertension in a Large Animal Model

Metabolic gene therapy in a canine with pulmonary hypertension secondary to degenerative mitral valve disease

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