CRISPR medicine for blood disorders: Progress and challenges in delivery
Blood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical proof to treat different blood disorders. Gen...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-01-01
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| Series: | Frontiers in Genome Editing |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fgeed.2022.1037290/full |
| _version_ | 1797959830322806784 |
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| author | Tahereh Mohammadian Gol Guillermo Ureña-Bailén Yujuan Hou Ralph Sinn Justin S. Antony Rupert Handgretinger Rupert Handgretinger Markus Mezger |
| author_facet | Tahereh Mohammadian Gol Guillermo Ureña-Bailén Yujuan Hou Ralph Sinn Justin S. Antony Rupert Handgretinger Rupert Handgretinger Markus Mezger |
| author_sort | Tahereh Mohammadian Gol |
| collection | DOAJ |
| description | Blood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical proof to treat different blood disorders. Genome editing components such as Cas nucleases, guide RNAs and base editors are supplied in the form of either a plasmid, an mRNA, or a ribonucleoprotein complex. The most common delivery vehicles for such components include viral vectors (e.g., AAVs and RV), non-viral vectors (e.g., LNPs and polymers) and physical delivery methods (e.g., electroporation and microinjection). Each of the delivery vehicles specified above has its own advantages and disadvantages and the development of a safe transferring method for ex vivo and in vivo application of genome editing components is still a big challenge. Moreover, the delivery of genome editing payload to the target blood cells possess key challenges to provide a possible cure for patients with inherited monogenic blood diseases and hematological neoplastic tumors. Here, we critically review and summarize the progress and challenges related to the delivery of genome editing elements to relevant blood cells in an ex vivo or in vivo setting. In addition, we have attempted to provide a future clinical perspective of genome editing to treat blood disorders with possible clinical grade improvements in delivery methods. |
| first_indexed | 2024-04-11T00:37:15Z |
| format | Article |
| id | doaj.art-af6536b6e18e46e6acc0fbb918946857 |
| institution | Directory Open Access Journal |
| issn | 2673-3439 |
| language | English |
| last_indexed | 2024-04-11T00:37:15Z |
| publishDate | 2023-01-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Genome Editing |
| spelling | doaj.art-af6536b6e18e46e6acc0fbb9189468572023-01-06T16:57:55ZengFrontiers Media S.A.Frontiers in Genome Editing2673-34392023-01-01410.3389/fgeed.2022.10372901037290CRISPR medicine for blood disorders: Progress and challenges in deliveryTahereh Mohammadian Gol0Guillermo Ureña-Bailén1Yujuan Hou2Ralph Sinn3Justin S. Antony4Rupert Handgretinger5Rupert Handgretinger6Markus Mezger7Department of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyAbu Dhabi Stem Cells Center, Abu Dhabi, United Arab EmiratesDepartment of Hematology and Oncology, University Children’s Hospital, University of Tübingen, Tübingen, GermanyBlood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical proof to treat different blood disorders. Genome editing components such as Cas nucleases, guide RNAs and base editors are supplied in the form of either a plasmid, an mRNA, or a ribonucleoprotein complex. The most common delivery vehicles for such components include viral vectors (e.g., AAVs and RV), non-viral vectors (e.g., LNPs and polymers) and physical delivery methods (e.g., electroporation and microinjection). Each of the delivery vehicles specified above has its own advantages and disadvantages and the development of a safe transferring method for ex vivo and in vivo application of genome editing components is still a big challenge. Moreover, the delivery of genome editing payload to the target blood cells possess key challenges to provide a possible cure for patients with inherited monogenic blood diseases and hematological neoplastic tumors. Here, we critically review and summarize the progress and challenges related to the delivery of genome editing elements to relevant blood cells in an ex vivo or in vivo setting. In addition, we have attempted to provide a future clinical perspective of genome editing to treat blood disorders with possible clinical grade improvements in delivery methods.https://www.frontiersin.org/articles/10.3389/fgeed.2022.1037290/fullCRISPR/Casblood disorderphysical deliveryviral vectorsnon-viral vectors |
| spellingShingle | Tahereh Mohammadian Gol Guillermo Ureña-Bailén Yujuan Hou Ralph Sinn Justin S. Antony Rupert Handgretinger Rupert Handgretinger Markus Mezger CRISPR medicine for blood disorders: Progress and challenges in delivery Frontiers in Genome Editing CRISPR/Cas blood disorder physical delivery viral vectors non-viral vectors |
| title | CRISPR medicine for blood disorders: Progress and challenges in delivery |
| title_full | CRISPR medicine for blood disorders: Progress and challenges in delivery |
| title_fullStr | CRISPR medicine for blood disorders: Progress and challenges in delivery |
| title_full_unstemmed | CRISPR medicine for blood disorders: Progress and challenges in delivery |
| title_short | CRISPR medicine for blood disorders: Progress and challenges in delivery |
| title_sort | crispr medicine for blood disorders progress and challenges in delivery |
| topic | CRISPR/Cas blood disorder physical delivery viral vectors non-viral vectors |
| url | https://www.frontiersin.org/articles/10.3389/fgeed.2022.1037290/full |
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