Delivering on the promise of gene editing for cystic fibrosis
In this review, we describe a path for translation of gene editing into therapy for cystic fibrosis (CF). Cystic fibrosis results from mutations in the CFTR gene, with one allele predominant in patient populations. This simple, genetic etiology makes gene editing appealing for treatment of this dise...
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| Format: | Article |
| Language: | English |
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KeAi Communications Co., Ltd.
2019-06-01
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| Series: | Genes and Diseases |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304218301363 |
| _version_ | 1827267051501125632 |
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| author | Craig A. Hodges Ronald A. Conlon |
| author_facet | Craig A. Hodges Ronald A. Conlon |
| author_sort | Craig A. Hodges |
| collection | DOAJ |
| description | In this review, we describe a path for translation of gene editing into therapy for cystic fibrosis (CF). Cystic fibrosis results from mutations in the CFTR gene, with one allele predominant in patient populations. This simple, genetic etiology makes gene editing appealing for treatment of this disease. There already have been success in applying this approach to cystic fibrosis in cell and animal models, although these advances have been modest in comparison to advances for other disease.Less than six years after its first demonstration in animals, CRISPR/Cas gene editing is in early clinical trials for several disorders. Most clinical trials, thus far, attempt to edit genes in cells of the blood lineages. The advantage of the blood is that the stem cells are known, can be isolated, edited, selected, expanded, and returned to the body. The likely next trials will be in the liver, which is accessible to many delivery methods. For cystic fibrosis, the biggest hurdle is to deliver editors to other, less accessible organs. We outline a path by which delivery can be improved.The translation of new therapies doesn't occur in isolation, and the development of gene editors is occurring as advances in gene therapy and small molecule therapeutics are being made. The advances made in gene therapy may help develop delivery vehicles for gene editing, although major improvements are needed. Conversely, the approval of effective small molecule therapies for many patients with cystic fibrosis will raise the bar for translation of gene editing. Keywords: CFTR gene, CRISPR/Cas9, Cystic fibrosis, Gene editing, Gene therapy |
| first_indexed | 2024-03-12T06:06:12Z |
| format | Article |
| id | doaj.art-e1f8e74ab66a44929f54c5e143807cfc |
| institution | Directory Open Access Journal |
| issn | 2352-3042 |
| language | English |
| last_indexed | 2025-03-22T04:27:36Z |
| publishDate | 2019-06-01 |
| publisher | KeAi Communications Co., Ltd. |
| record_format | Article |
| series | Genes and Diseases |
| spelling | doaj.art-e1f8e74ab66a44929f54c5e143807cfc2024-04-28T05:30:13ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422019-06-016297108Delivering on the promise of gene editing for cystic fibrosisCraig A. Hodges0Ronald A. Conlon1Department of Pediatrics, Case Western Reserve University, Cleveland, OH, USA; Department of Genetics and Genome Sciences, Case Western Reserve University, Cleveland, OH, USADepartment of Genetics and Genome Sciences, Case Western Reserve University, Cleveland, OH, USA; Corresponding author. Case Western Reserve University, Biomedical Research Building 623, 10900 Euclid Avenue Cleveland, OH 44106-4955, USA.In this review, we describe a path for translation of gene editing into therapy for cystic fibrosis (CF). Cystic fibrosis results from mutations in the CFTR gene, with one allele predominant in patient populations. This simple, genetic etiology makes gene editing appealing for treatment of this disease. There already have been success in applying this approach to cystic fibrosis in cell and animal models, although these advances have been modest in comparison to advances for other disease.Less than six years after its first demonstration in animals, CRISPR/Cas gene editing is in early clinical trials for several disorders. Most clinical trials, thus far, attempt to edit genes in cells of the blood lineages. The advantage of the blood is that the stem cells are known, can be isolated, edited, selected, expanded, and returned to the body. The likely next trials will be in the liver, which is accessible to many delivery methods. For cystic fibrosis, the biggest hurdle is to deliver editors to other, less accessible organs. We outline a path by which delivery can be improved.The translation of new therapies doesn't occur in isolation, and the development of gene editors is occurring as advances in gene therapy and small molecule therapeutics are being made. The advances made in gene therapy may help develop delivery vehicles for gene editing, although major improvements are needed. Conversely, the approval of effective small molecule therapies for many patients with cystic fibrosis will raise the bar for translation of gene editing. Keywords: CFTR gene, CRISPR/Cas9, Cystic fibrosis, Gene editing, Gene therapyhttp://www.sciencedirect.com/science/article/pii/S2352304218301363 |
| spellingShingle | Craig A. Hodges Ronald A. Conlon Delivering on the promise of gene editing for cystic fibrosis Genes and Diseases |
| title | Delivering on the promise of gene editing for cystic fibrosis |
| title_full | Delivering on the promise of gene editing for cystic fibrosis |
| title_fullStr | Delivering on the promise of gene editing for cystic fibrosis |
| title_full_unstemmed | Delivering on the promise of gene editing for cystic fibrosis |
| title_short | Delivering on the promise of gene editing for cystic fibrosis |
| title_sort | delivering on the promise of gene editing for cystic fibrosis |
| url | http://www.sciencedirect.com/science/article/pii/S2352304218301363 |
| work_keys_str_mv | AT craigahodges deliveringonthepromiseofgeneeditingforcysticfibrosis AT ronaldaconlon deliveringonthepromiseofgeneeditingforcysticfibrosis |