Genome Editing for Cystic Fibrosis
Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the CF Transmembrane-conductance Regulator gene (<i>CFTR</i>). Remarkable progress in basic research has led to the discovery of highly effective CFTR modulators. Now ~90% of CF patients are treatable....
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-06-01
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| Series: | Cells |
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| Online Access: | https://www.mdpi.com/2073-4409/12/12/1555 |
| _version_ | 1797595568893067264 |
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| author | Guoshun Wang |
| author_facet | Guoshun Wang |
| author_sort | Guoshun Wang |
| collection | DOAJ |
| description | Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the CF Transmembrane-conductance Regulator gene (<i>CFTR</i>). Remarkable progress in basic research has led to the discovery of highly effective CFTR modulators. Now ~90% of CF patients are treatable. However, these modulator therapies are not curative and do not cover the full spectrum of <i>CFTR</i> mutations. Thus, there is a continued need to develop a complete and durable therapy that can treat all CF patients once and for all. As CF is a genetic disease, the ultimate therapy would be in-situ repair of the genetic lesions in the genome. Within the past few years, new technologies, such as CRISPR/Cas gene editing, have emerged as an appealing platform to revise the genome, ushering in a new era of genetic therapy. This review provided an update on this rapidly evolving field and the status of adapting the technology for CF therapy. |
| first_indexed | 2024-03-11T02:39:23Z |
| format | Article |
| id | doaj.art-006630b8ac3340449bdc8803cdc23f3d |
| institution | Directory Open Access Journal |
| issn | 2073-4409 |
| language | English |
| last_indexed | 2024-03-11T02:39:23Z |
| publishDate | 2023-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cells |
| spelling | doaj.art-006630b8ac3340449bdc8803cdc23f3d2023-11-18T09:42:40ZengMDPI AGCells2073-44092023-06-011212155510.3390/cells12121555Genome Editing for Cystic FibrosisGuoshun Wang0Department of Microbiology, Immunology and Parasitology, Louisiana State University Health Sciences Center, CSRB 607, 533 Bolivar Street, New Orleans, LA 70112, USACystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the CF Transmembrane-conductance Regulator gene (<i>CFTR</i>). Remarkable progress in basic research has led to the discovery of highly effective CFTR modulators. Now ~90% of CF patients are treatable. However, these modulator therapies are not curative and do not cover the full spectrum of <i>CFTR</i> mutations. Thus, there is a continued need to develop a complete and durable therapy that can treat all CF patients once and for all. As CF is a genetic disease, the ultimate therapy would be in-situ repair of the genetic lesions in the genome. Within the past few years, new technologies, such as CRISPR/Cas gene editing, have emerged as an appealing platform to revise the genome, ushering in a new era of genetic therapy. This review provided an update on this rapidly evolving field and the status of adapting the technology for CF therapy.https://www.mdpi.com/2073-4409/12/12/1555cystic fibrosisCRISPR/Casgenome editinggene editingCFTR |
| spellingShingle | Guoshun Wang Genome Editing for Cystic Fibrosis Cells cystic fibrosis CRISPR/Cas genome editing gene editing CFTR |
| title | Genome Editing for Cystic Fibrosis |
| title_full | Genome Editing for Cystic Fibrosis |
| title_fullStr | Genome Editing for Cystic Fibrosis |
| title_full_unstemmed | Genome Editing for Cystic Fibrosis |
| title_short | Genome Editing for Cystic Fibrosis |
| title_sort | genome editing for cystic fibrosis |
| topic | cystic fibrosis CRISPR/Cas genome editing gene editing CFTR |
| url | https://www.mdpi.com/2073-4409/12/12/1555 |
| work_keys_str_mv | AT guoshunwang genomeeditingforcysticfibrosis |