Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing
Since the early days of its conceptualization and application, human gene transfer held the promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field went through alternated periods of enthusiasm and distrust. The development of refined technologies allowing site...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-05-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/21/11/3903 |
| _version_ | 1797566635069931520 |
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| author | Giulia Maule Daniele Arosio Anna Cereseto |
| author_facet | Giulia Maule Daniele Arosio Anna Cereseto |
| author_sort | Giulia Maule |
| collection | DOAJ |
| description | Since the early days of its conceptualization and application, human gene transfer held the promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field went through alternated periods of enthusiasm and distrust. The development of refined technologies allowing site specific modification with programmable nucleases highly revived the gene therapy field. CRISPR nucleases and derived technologies tremendously facilitate genome manipulation offering diversified strategies to reverse mutations. Here we discuss the advancement of gene therapy, from therapeutic nucleic acids to genome editing techniques, designed to reverse genetic defects in CF. We provide a roadmap through technologies and strategies tailored to correct different types of mutations in the cystic fibrosis transmembrane regulator (<i>CFTR)</i> gene, and their applications for the development of experimental models valuable for the advancement of CF therapies. |
| first_indexed | 2024-03-10T19:30:13Z |
| format | Article |
| id | doaj.art-fc238cd8ff1d4549b9253f3f83a699e4 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T19:30:13Z |
| publishDate | 2020-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-fc238cd8ff1d4549b9253f3f83a699e42023-11-20T02:16:30ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672020-05-012111390310.3390/ijms21113903Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome EditingGiulia Maule0Daniele Arosio1Anna Cereseto2Department of Cellular Computational Integrative Biology (CIBIO), University of Trento, 38123 Trento, ItalyNational Council of Research, CNR, 38123 Trento, ItalyDepartment of Cellular Computational Integrative Biology (CIBIO), University of Trento, 38123 Trento, ItalySince the early days of its conceptualization and application, human gene transfer held the promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field went through alternated periods of enthusiasm and distrust. The development of refined technologies allowing site specific modification with programmable nucleases highly revived the gene therapy field. CRISPR nucleases and derived technologies tremendously facilitate genome manipulation offering diversified strategies to reverse mutations. Here we discuss the advancement of gene therapy, from therapeutic nucleic acids to genome editing techniques, designed to reverse genetic defects in CF. We provide a roadmap through technologies and strategies tailored to correct different types of mutations in the cystic fibrosis transmembrane regulator (<i>CFTR)</i> gene, and their applications for the development of experimental models valuable for the advancement of CF therapies.https://www.mdpi.com/1422-0067/21/11/3903programmable nucleasesCRISPR-Casgenome editing |
| spellingShingle | Giulia Maule Daniele Arosio Anna Cereseto Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing International Journal of Molecular Sciences programmable nucleases CRISPR-Cas genome editing |
| title | Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing |
| title_full | Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing |
| title_fullStr | Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing |
| title_full_unstemmed | Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing |
| title_short | Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing |
| title_sort | gene therapy for cystic fibrosis progress and challenges of genome editing |
| topic | programmable nucleases CRISPR-Cas genome editing |
| url | https://www.mdpi.com/1422-0067/21/11/3903 |
| work_keys_str_mv | AT giuliamaule genetherapyforcysticfibrosisprogressandchallengesofgenomeediting AT danielearosio genetherapyforcysticfibrosisprogressandchallengesofgenomeediting AT annacereseto genetherapyforcysticfibrosisprogressandchallengesofgenomeediting |