Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9
Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods I...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Lu, Pei-jie [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2023 |
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| Anmerkung: |
© Huazhong University of Science and Technology 2023 |
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| Übergeordnetes Werk: |
Enthalten in: Journal of Huazhong University of Science and Technology - Wuhan : Huazhong Univ. of Science and Technology, 2002, 43(2023), 2 vom: 18. März, Seite 261-267 |
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| Übergeordnetes Werk: |
volume:43 ; year:2023 ; number:2 ; day:18 ; month:03 ; pages:261-267 |
| Links: |
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| DOI / URN: |
10.1007/s11596-023-2707-8 |
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| Katalog-ID: |
SPR050198238 |
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| 520 | |a Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. | ||
| 700 | 1 | |a Zhang, Pei |4 aut | |
| 700 | 1 | |a Liu, Yu-chun |4 aut | |
| 700 | 1 | |a Jing, Na |4 aut | |
| 700 | 1 | |a Guo, Ya-nan |4 aut | |
| 700 | 1 | |a Wang, Peng-shuai |4 aut | |
| 700 | 1 | |a Su, Lin-lin |4 aut | |
| 700 | 1 | |a Guo, Qi |4 aut | |
| 700 | 1 | |a Ma, Qiang |4 aut | |
| 700 | 1 | |a Xu, Yu-ming |4 aut | |
| 700 | 1 | |a Zhang, Shou-tao |4 aut | |
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10.1007/s11596-023-2707-8 doi (DE-627)SPR050198238 (SPR)s11596-023-2707-8-e DE-627 ger DE-627 rakwb eng Lu, Pei-jie verfasserin aut Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Huazhong University of Science and Technology 2023 Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. Zhang, Pei aut Liu, Yu-chun aut Jing, Na aut Guo, Ya-nan aut Wang, Peng-shuai aut Su, Lin-lin aut Guo, Qi aut Ma, Qiang aut Xu, Yu-ming aut Zhang, Shou-tao aut Enthalten in Journal of Huazhong University of Science and Technology Wuhan : Huazhong Univ. of Science and Technology, 2002 43(2023), 2 vom: 18. März, Seite 261-267 (DE-627)537444408 (DE-600)2376511-2 1993-1352 nnns volume:43 year:2023 number:2 day:18 month:03 pages:261-267 https://dx.doi.org/10.1007/s11596-023-2707-8 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER GBV_ILN_11 GBV_ILN_32 GBV_ILN_120 GBV_ILN_121 GBV_ILN_374 GBV_ILN_2700 GBV_ILN_2817 AR 43 2023 2 18 03 261-267 |
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10.1007/s11596-023-2707-8 doi (DE-627)SPR050198238 (SPR)s11596-023-2707-8-e DE-627 ger DE-627 rakwb eng Lu, Pei-jie verfasserin aut Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Huazhong University of Science and Technology 2023 Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. Zhang, Pei aut Liu, Yu-chun aut Jing, Na aut Guo, Ya-nan aut Wang, Peng-shuai aut Su, Lin-lin aut Guo, Qi aut Ma, Qiang aut Xu, Yu-ming aut Zhang, Shou-tao aut Enthalten in Journal of Huazhong University of Science and Technology Wuhan : Huazhong Univ. of Science and Technology, 2002 43(2023), 2 vom: 18. März, Seite 261-267 (DE-627)537444408 (DE-600)2376511-2 1993-1352 nnns volume:43 year:2023 number:2 day:18 month:03 pages:261-267 https://dx.doi.org/10.1007/s11596-023-2707-8 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER GBV_ILN_11 GBV_ILN_32 GBV_ILN_120 GBV_ILN_121 GBV_ILN_374 GBV_ILN_2700 GBV_ILN_2817 AR 43 2023 2 18 03 261-267 |
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10.1007/s11596-023-2707-8 doi (DE-627)SPR050198238 (SPR)s11596-023-2707-8-e DE-627 ger DE-627 rakwb eng Lu, Pei-jie verfasserin aut Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Huazhong University of Science and Technology 2023 Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. Zhang, Pei aut Liu, Yu-chun aut Jing, Na aut Guo, Ya-nan aut Wang, Peng-shuai aut Su, Lin-lin aut Guo, Qi aut Ma, Qiang aut Xu, Yu-ming aut Zhang, Shou-tao aut Enthalten in Journal of Huazhong University of Science and Technology Wuhan : Huazhong Univ. of Science and Technology, 2002 43(2023), 2 vom: 18. März, Seite 261-267 (DE-627)537444408 (DE-600)2376511-2 1993-1352 nnns volume:43 year:2023 number:2 day:18 month:03 pages:261-267 https://dx.doi.org/10.1007/s11596-023-2707-8 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER GBV_ILN_11 GBV_ILN_32 GBV_ILN_120 GBV_ILN_121 GBV_ILN_374 GBV_ILN_2700 GBV_ILN_2817 AR 43 2023 2 18 03 261-267 |
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10.1007/s11596-023-2707-8 doi (DE-627)SPR050198238 (SPR)s11596-023-2707-8-e DE-627 ger DE-627 rakwb eng Lu, Pei-jie verfasserin aut Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Huazhong University of Science and Technology 2023 Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. Zhang, Pei aut Liu, Yu-chun aut Jing, Na aut Guo, Ya-nan aut Wang, Peng-shuai aut Su, Lin-lin aut Guo, Qi aut Ma, Qiang aut Xu, Yu-ming aut Zhang, Shou-tao aut Enthalten in Journal of Huazhong University of Science and Technology Wuhan : Huazhong Univ. of Science and Technology, 2002 43(2023), 2 vom: 18. März, Seite 261-267 (DE-627)537444408 (DE-600)2376511-2 1993-1352 nnns volume:43 year:2023 number:2 day:18 month:03 pages:261-267 https://dx.doi.org/10.1007/s11596-023-2707-8 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER GBV_ILN_11 GBV_ILN_32 GBV_ILN_120 GBV_ILN_121 GBV_ILN_374 GBV_ILN_2700 GBV_ILN_2817 AR 43 2023 2 18 03 261-267 |
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10.1007/s11596-023-2707-8 doi (DE-627)SPR050198238 (SPR)s11596-023-2707-8-e DE-627 ger DE-627 rakwb eng Lu, Pei-jie verfasserin aut Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 2023 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © Huazhong University of Science and Technology 2023 Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. Zhang, Pei aut Liu, Yu-chun aut Jing, Na aut Guo, Ya-nan aut Wang, Peng-shuai aut Su, Lin-lin aut Guo, Qi aut Ma, Qiang aut Xu, Yu-ming aut Zhang, Shou-tao aut Enthalten in Journal of Huazhong University of Science and Technology Wuhan : Huazhong Univ. of Science and Technology, 2002 43(2023), 2 vom: 18. März, Seite 261-267 (DE-627)537444408 (DE-600)2376511-2 1993-1352 nnns volume:43 year:2023 number:2 day:18 month:03 pages:261-267 https://dx.doi.org/10.1007/s11596-023-2707-8 lizenzpflichtig Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER GBV_ILN_11 GBV_ILN_32 GBV_ILN_120 GBV_ILN_121 GBV_ILN_374 GBV_ILN_2700 GBV_ILN_2817 AR 43 2023 2 18 03 261-267 |
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Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 |
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Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 |
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Lu, Pei-jie |
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Lu, Pei-jie Zhang, Pei Liu, Yu-chun Jing, Na Guo, Ya-nan Wang, Peng-shuai Su, Lin-lin Guo, Qi Ma, Qiang Xu, Yu-ming Zhang, Shou-tao |
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gene repair of ipsc line with gars (g294r) mutation of cmt2d disease by crispr/cas9 |
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Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 |
| abstract |
Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. © Huazhong University of Science and Technology 2023 |
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Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. © Huazhong University of Science and Technology 2023 |
| abstract_unstemmed |
Objective Charcot-Marie-Tooth disease (CMT) severely affects patient activity, and may cause disability. However, no clinical treatment is available to reverse the disease course. The combination of CRISPR/Cas9 and iPSCs may have therapeutic potential against nervous diseases, such as CMT. Methods In the present study, the skin fibroblasts of CMT type 2D (CMT2D) patients with the c.880G>A heterozygous nucleotide mutation in the GARS gene were reprogrammed into iPSCs using three plasmids (pCXLE-hSK, pCXLE-hUL and pCXLE-hOCT3/4-shp5-F). Then, CRISPR/Cas9 technology was used to repair the mutated gene sites at the iPSC level. Results An iPSC line derived from the GARS (G294R) family with fibular atrophy was successfully induced, and the mutated gene loci were repaired at the iPSC level using CRISPR/Cas9 technology. These findings lay the foundation for future research on drug screening and cell therapy. Conclusion iPSCs can differentiate into different cell types, and originate from autologous cells. Therefore, they are promising for the development of autologous cell therapies for degenerative diseases. The combination of CRISPR/Cas9 and iPSCs may open a new avenue for the treatment of nervous diseases, such as CMT. © Huazhong University of Science and Technology 2023 |
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Gene Repair of iPSC Line with GARS (G294R) Mutation of CMT2D Disease by CRISPR/Cas9 |
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