Prime Editing for Human Gene Therapy: Where Are We Now?
Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-02-01
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| Series: | Cells |
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| Online Access: | https://www.mdpi.com/2073-4409/12/4/536 |
| _version_ | 1797621819803435008 |
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| author | Kelly Godbout Jacques P. Tremblay |
| author_facet | Kelly Godbout Jacques P. Tremblay |
| author_sort | Kelly Godbout |
| collection | DOAJ |
| description | Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way for the development of those therapies. Improvement of this system led to the recent development of an outstanding technology called prime editing. This system can introduce targeted insertions, deletions, and all 12 possible base-to-base conversions in the human genome. Since the first publication on prime editing in 2019, groups all around the world have worked on this promising technology to develop a treatment for genetic diseases. To date, prime editing has been attempted in preclinical studies for liver, eye, skin, muscular, and neurodegenerative hereditary diseases, in addition to cystic fibrosis, beta-thalassemia, X-linked severe combined immunodeficiency, and cancer. In this review, we portrayed where we are now on prime editing for human gene therapy and outlined the best strategies for correcting pathogenic mutations by prime editing. |
| first_indexed | 2024-03-11T09:01:25Z |
| format | Article |
| id | doaj.art-e55428c35e57448398c4fcad8f166204 |
| institution | Directory Open Access Journal |
| issn | 2073-4409 |
| language | English |
| last_indexed | 2024-03-11T09:01:25Z |
| publishDate | 2023-02-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Cells |
| spelling | doaj.art-e55428c35e57448398c4fcad8f1662042023-11-16T19:43:42ZengMDPI AGCells2073-44092023-02-0112453610.3390/cells12040536Prime Editing for Human Gene Therapy: Where Are We Now?Kelly Godbout0Jacques P. Tremblay1Centre de Recherche du CHU de Québec-Université Laval, Quebec City, QC G1V 4G2, CanadaCentre de Recherche du CHU de Québec-Université Laval, Quebec City, QC G1V 4G2, CanadaGene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way for the development of those therapies. Improvement of this system led to the recent development of an outstanding technology called prime editing. This system can introduce targeted insertions, deletions, and all 12 possible base-to-base conversions in the human genome. Since the first publication on prime editing in 2019, groups all around the world have worked on this promising technology to develop a treatment for genetic diseases. To date, prime editing has been attempted in preclinical studies for liver, eye, skin, muscular, and neurodegenerative hereditary diseases, in addition to cystic fibrosis, beta-thalassemia, X-linked severe combined immunodeficiency, and cancer. In this review, we portrayed where we are now on prime editing for human gene therapy and outlined the best strategies for correcting pathogenic mutations by prime editing.https://www.mdpi.com/2073-4409/12/4/536prime editinggene therapyinherited diseasesgenetic diseasesCRISPR/Cas9 |
| spellingShingle | Kelly Godbout Jacques P. Tremblay Prime Editing for Human Gene Therapy: Where Are We Now? Cells prime editing gene therapy inherited diseases genetic diseases CRISPR/Cas9 |
| title | Prime Editing for Human Gene Therapy: Where Are We Now? |
| title_full | Prime Editing for Human Gene Therapy: Where Are We Now? |
| title_fullStr | Prime Editing for Human Gene Therapy: Where Are We Now? |
| title_full_unstemmed | Prime Editing for Human Gene Therapy: Where Are We Now? |
| title_short | Prime Editing for Human Gene Therapy: Where Are We Now? |
| title_sort | prime editing for human gene therapy where are we now |
| topic | prime editing gene therapy inherited diseases genetic diseases CRISPR/Cas9 |
| url | https://www.mdpi.com/2073-4409/12/4/536 |
| work_keys_str_mv | AT kellygodbout primeeditingforhumangenetherapywherearewenow AT jacquesptremblay primeeditingforhumangenetherapywherearewenow |