Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects
Genome editing has recently evolved from a theoretical concept to a powerful and versatile set of tools. The discovery and implementation of CRISPR-Cas9 technology have propelled the field further into a new era. This RNA-guided system allows for specific modification of target genes, offering high...
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| Format: | Article |
| Language: | English |
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Cambridge University Press
2025-01-01
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| Series: | Expert Reviews in Molecular Medicine |
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| Online Access: | https://www.cambridge.org/core/product/identifier/S1462399425000109/type/journal_article |
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| author | Busra Cetin Fulya Erendor Yunus Emre Eksi Ahter D. Sanlioglu Salih Sanlioglu |
| author_facet | Busra Cetin Fulya Erendor Yunus Emre Eksi Ahter D. Sanlioglu Salih Sanlioglu |
| author_sort | Busra Cetin |
| collection | DOAJ |
| description | Genome editing has recently evolved from a theoretical concept to a powerful and versatile set of tools. The discovery and implementation of CRISPR-Cas9 technology have propelled the field further into a new era. This RNA-guided system allows for specific modification of target genes, offering high accuracy and efficiency. Encouraging results are being announced in clinical trials employed in conditions like sickle cell disease (SCD) and transfusion-dependent beta-thalassaemia (TDT). The path finally led the way to the recent FDA approval of the first gene therapy drug utilising the CRISPR/Cas9 system to edit autologous CD34+ haematopoietic stem cells in SCD patients (Casgevy). Ongoing research explores the potential of CRISPR technology for cancer therapies, HIV treatment and other complex diseases. Despite its remarkable potential, CRISPR technology faces challenges such as off-target effects, suboptimal delivery systems, long-term safety concerns, scalability, ethical dilemmas and potential repercussions of genetic alterations, particularly in the case of germline editing. Here, we examine the transformative role of CRISPR technologies, including base editing and prime editing approaches, in modifying the genetic and epigenetic codes in the human genome and provide a comprehensive focus, particularly on relevant clinical applications, to unlock the full potential and challenges of gene editing. |
| format | Article |
| id | doaj-art-2b049c8c98c64bafbcb7c9e36136d064 |
| institution | Kabale University |
| issn | 1462-3994 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Cambridge University Press |
| record_format | Article |
| series | Expert Reviews in Molecular Medicine |
| spelling | doaj-art-2b049c8c98c64bafbcb7c9e36136d0642025-08-20T12:57:28ZengCambridge University PressExpert Reviews in Molecular Medicine1462-39942025-01-012710.1017/erm.2025.10Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospectsBusra Cetin0https://orcid.org/0000-0002-9644-7610Fulya Erendor1Yunus Emre Eksi2Ahter D. Sanlioglu3Salih Sanlioglu4https://orcid.org/0000-0001-8379-5678Department of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, TurkeyDepartment of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, TurkeyDepartment of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, TurkeyDepartment of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, TurkeyDepartment of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, TurkeyGenome editing has recently evolved from a theoretical concept to a powerful and versatile set of tools. The discovery and implementation of CRISPR-Cas9 technology have propelled the field further into a new era. This RNA-guided system allows for specific modification of target genes, offering high accuracy and efficiency. Encouraging results are being announced in clinical trials employed in conditions like sickle cell disease (SCD) and transfusion-dependent beta-thalassaemia (TDT). The path finally led the way to the recent FDA approval of the first gene therapy drug utilising the CRISPR/Cas9 system to edit autologous CD34+ haematopoietic stem cells in SCD patients (Casgevy). Ongoing research explores the potential of CRISPR technology for cancer therapies, HIV treatment and other complex diseases. Despite its remarkable potential, CRISPR technology faces challenges such as off-target effects, suboptimal delivery systems, long-term safety concerns, scalability, ethical dilemmas and potential repercussions of genetic alterations, particularly in the case of germline editing. Here, we examine the transformative role of CRISPR technologies, including base editing and prime editing approaches, in modifying the genetic and epigenetic codes in the human genome and provide a comprehensive focus, particularly on relevant clinical applications, to unlock the full potential and challenges of gene editing.https://www.cambridge.org/core/product/identifier/S1462399425000109/type/journal_articleCRISPR/Casgenetic diseasesgene editinggene therapymedical genetics |
| spellingShingle | Busra Cetin Fulya Erendor Yunus Emre Eksi Ahter D. Sanlioglu Salih Sanlioglu Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects Expert Reviews in Molecular Medicine CRISPR/Cas genetic diseases gene editing gene therapy medical genetics |
| title | Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects |
| title_full | Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects |
| title_fullStr | Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects |
| title_full_unstemmed | Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects |
| title_short | Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects |
| title_sort | advancing crispr genome editing into gene therapy clinical trials progress and future prospects |
| topic | CRISPR/Cas genetic diseases gene editing gene therapy medical genetics |
| url | https://www.cambridge.org/core/product/identifier/S1462399425000109/type/journal_article |
| work_keys_str_mv | AT busracetin advancingcrisprgenomeeditingintogenetherapyclinicaltrialsprogressandfutureprospects AT fulyaerendor advancingcrisprgenomeeditingintogenetherapyclinicaltrialsprogressandfutureprospects AT yunusemreeksi advancingcrisprgenomeeditingintogenetherapyclinicaltrialsprogressandfutureprospects AT ahterdsanlioglu advancingcrisprgenomeeditingintogenetherapyclinicaltrialsprogressandfutureprospects AT salihsanlioglu advancingcrisprgenomeeditingintogenetherapyclinicaltrialsprogressandfutureprospects |