On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis
Cystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking. Next generation biotechnologies and more efficient cell-based and in vivo disease models are accelerating the development of novel therapies for CF. Gene editing tools, like CRISPR-based systems, can be u...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2021-04-01
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| Series: | Frontiers in Pharmacology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2021.662110/full |
| _version_ | 1831592128756580352 |
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| author | Marjolein Ensinck Angélique Mottais Claire Detry Teresinha Leal Marianne S. Carlon |
| author_facet | Marjolein Ensinck Angélique Mottais Claire Detry Teresinha Leal Marianne S. Carlon |
| author_sort | Marjolein Ensinck |
| collection | DOAJ |
| description | Cystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking. Next generation biotechnologies and more efficient cell-based and in vivo disease models are accelerating the development of novel therapies for CF. Gene editing tools, like CRISPR-based systems, can be used to make targeted modifications in the genome, allowing to correct mutations directly in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Alternatively, with these tools more relevant disease models can be generated, which in turn will be invaluable to evaluate novel gene editing-based therapies for CF. This critical review offers a comprehensive description of currently available tools for genome editing, and the cell and animal models which are available to evaluate them. Next, we will give an extensive overview of proof-of-concept applications of gene editing in the field of CF. Finally, we will touch upon the challenges that need to be addressed before these proof-of-concept studies can be translated towards a therapy for people with CF. |
| first_indexed | 2024-12-18T01:37:55Z |
| format | Article |
| id | doaj.art-a82ba05bff824c6787cb254a4cbb1a28 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-12-18T01:37:55Z |
| publishDate | 2021-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-a82ba05bff824c6787cb254a4cbb1a282022-12-21T21:25:25ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122021-04-011210.3389/fphar.2021.662110662110On the Corner of Models and Cure: Gene Editing in Cystic FibrosisMarjolein Ensinck0Angélique Mottais1Claire Detry2Teresinha Leal3Marianne S. Carlon4Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, BelgiumInstitut de Recherche Expérimentale et Clinique, Louvain Centre for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, BelgiumInstitut de Recherche Expérimentale et Clinique, Louvain Centre for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, BelgiumInstitut de Recherche Expérimentale et Clinique, Louvain Centre for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, BelgiumMolecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, BelgiumCystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking. Next generation biotechnologies and more efficient cell-based and in vivo disease models are accelerating the development of novel therapies for CF. Gene editing tools, like CRISPR-based systems, can be used to make targeted modifications in the genome, allowing to correct mutations directly in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Alternatively, with these tools more relevant disease models can be generated, which in turn will be invaluable to evaluate novel gene editing-based therapies for CF. This critical review offers a comprehensive description of currently available tools for genome editing, and the cell and animal models which are available to evaluate them. Next, we will give an extensive overview of proof-of-concept applications of gene editing in the field of CF. Finally, we will touch upon the challenges that need to be addressed before these proof-of-concept studies can be translated towards a therapy for people with CF.https://www.frontiersin.org/articles/10.3389/fphar.2021.662110/fullcystic fibrosisCFTRgene therapygene editingCF cell modelsCF animal models |
| spellingShingle | Marjolein Ensinck Angélique Mottais Claire Detry Teresinha Leal Marianne S. Carlon On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis Frontiers in Pharmacology cystic fibrosis CFTR gene therapy gene editing CF cell models CF animal models |
| title | On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis |
| title_full | On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis |
| title_fullStr | On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis |
| title_full_unstemmed | On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis |
| title_short | On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis |
| title_sort | on the corner of models and cure gene editing in cystic fibrosis |
| topic | cystic fibrosis CFTR gene therapy gene editing CF cell models CF animal models |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2021.662110/full |
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