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Optimizations of In Vitro Mucus and Cell Culture Models to Better Predict In Vivo Gene Transfer in Pathological Lung Respiratory Airways: Cystic Fibrosis as an Example

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Univ Brest, INSERM, EFS, UMR 1078, GGB, F-29200 Brest, France
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IRDL UMR CNRS 6027, Université de Bretagne Occidentale, UFR Sciences et Techniques, 6, Avenue Victor Le Gorgeu CS 93837, CEDEX 3, 29238 Brest, France
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Centre de Ressources et de Compétences de la Mucoviscidose, Fondation Ildys, Presqu’île de Perharidy, 29680 Roscoff, France
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CHRU de Brest, Service de Génétique Médicale et Biologie de la Reproduction, Centre de Référence des Maladies Rares “Maladies Neuromusculaires”, F-29200 Brest, France
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Author to whom correspondence should be addressed.
Pharmaceutics 2021, 13(1), 47; https://doi.org/10.3390/pharmaceutics13010047
Received: 1 December 2020 / Revised: 22 December 2020 / Accepted: 28 December 2020 / Published: 31 December 2020
(This article belongs to the Section Gene and Cell Therapy)
The respiratory epithelium can be affected by many diseases that could be treated using aerosol gene therapy. Among these, cystic fibrosis (CF) is a lethal inherited disease characterized by airways complications, which determine the life expectancy and the effectiveness of aerosolized treatments. Beside evaluations performed under in vivo settings, cell culture models mimicking in vivo pathophysiological conditions can provide complementary insights into the potential of gene transfer strategies. Such models must consider multiple parameters, following the rationale that proper gene transfer evaluations depend on whether they are performed under experimental conditions close to pathophysiological settings. In addition, the mucus layer, which covers the epithelial cells, constitutes a physical barrier for gene delivery, especially in diseases such as CF. Artificial mucus models featuring physical and biological properties similar to CF mucus allow determining the ability of gene transfer systems to effectively reach the underlying epithelium. In this review, we describe mucus and cellular models relevant for CF aerosol gene therapy, with a particular emphasis on mucus rheology. We strongly believe that combining multiple pathophysiological features in single complex cell culture models could help bridge the gaps between in vitro and in vivo settings, as well as viral and non-viral gene delivery strategies. View Full-Text
Keywords: cystic fibrosis; gene delivery; in vitro model; mucus; airway epithelium cystic fibrosis; gene delivery; in vitro model; mucus; airway epithelium
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MDPI and ACS Style

Ghanem, R.; Laurent, V.; Roquefort, P.; Haute, T.; Ramel, S.; Le Gall, T.; Aubry, T.; Montier, T. Optimizations of In Vitro Mucus and Cell Culture Models to Better Predict In Vivo Gene Transfer in Pathological Lung Respiratory Airways: Cystic Fibrosis as an Example. Pharmaceutics 2021, 13, 47. https://doi.org/10.3390/pharmaceutics13010047

AMA Style

Ghanem R, Laurent V, Roquefort P, Haute T, Ramel S, Le Gall T, Aubry T, Montier T. Optimizations of In Vitro Mucus and Cell Culture Models to Better Predict In Vivo Gene Transfer in Pathological Lung Respiratory Airways: Cystic Fibrosis as an Example. Pharmaceutics. 2021; 13(1):47. https://doi.org/10.3390/pharmaceutics13010047

Chicago/Turabian Style

Ghanem, Rosy, Véronique Laurent, Philippe Roquefort, Tanguy Haute, Sophie Ramel, Tony Le Gall, Thierry Aubry, and Tristan Montier. 2021. "Optimizations of In Vitro Mucus and Cell Culture Models to Better Predict In Vivo Gene Transfer in Pathological Lung Respiratory Airways: Cystic Fibrosis as an Example" Pharmaceutics 13, no. 1: 47. https://doi.org/10.3390/pharmaceutics13010047

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