Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy
Advances in ex vivo technologies of human genome editing have made it possible to develop new approaches to the treatment of genetic, oncological, infectious and other diseases, which may involve the use of biomedical cell products. However, despite the rapid development of these technologies and a...
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| Format: | Article |
| Language: | Russian |
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Ministry of Health of the Russian Federation. Federal State Budgetary Institution «Scientific Centre for Expert Evaluation of Medicinal Products»
2018-09-01
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| Series: | Биопрепараты: Профилактика, диагностика, лечение |
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| Online Access: | https://www.biopreparations.ru/jour/article/view/169 |
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| author | A. A. Goryaev M. V. Savkina K. M. Mefed V. P. Bondarev V. A. Merkulov V. V. Tarasov |
| author_facet | A. A. Goryaev M. V. Savkina K. M. Mefed V. P. Bondarev V. A. Merkulov V. V. Tarasov |
| author_sort | A. A. Goryaev |
| collection | DOAJ |
| description | Advances in ex vivo technologies of human genome editing have made it possible to develop new approaches to the treatment of genetic, oncological, infectious and other diseases, which may involve the use of biomedical cell products. However, despite the rapid development of these technologies and a large number of clinical trials conducted in many countries around the world, only 4 products (Strimvelis, Zalmoxis, Kymriah and Yescarta) containing ex vivo genetically modified human cells are authorised for use in the European Union and the United States of America. This paper considers three promising technologies (ZFN, TALEN and CRISPR) that allow for easy and effective editing of the genome at the sites of interest, thereby creating a platform for further development of the genetic engineering of human cells. It describes the technology of engineering chimeric antigen receptors (CARs). It also provides data on the efficacy and safety of the approved products: Strimvelis which contains autologous CD34+ cells transduced ex vivo with a retroviral vector containing adenosine deaminase gene, Zalmoxis which contains modified allogeneic T-cells, and two products: Kymriah and Yescarta which contain autologous T-cells with CARs to CD19 antigen, intended for the treatment of CD19+ hematological malignancies. |
| format | Article |
| id | doaj-art-8d31812f168f4b598133faa1ea672afe |
| institution | Kabale University |
| issn | 2221-996X 2619-1156 |
| language | Russian |
| publishDate | 2018-09-01 |
| publisher | Ministry of Health of the Russian Federation. Federal State Budgetary Institution «Scientific Centre for Expert Evaluation of Medicinal Products» |
| record_format | Article |
| series | Биопрепараты: Профилактика, диагностика, лечение |
| spelling | doaj-art-8d31812f168f4b598133faa1ea672afe2025-08-20T03:43:15ZrusMinistry of Health of the Russian Federation. Federal State Budgetary Institution «Scientific Centre for Expert Evaluation of Medicinal Products»Биопрепараты: Профилактика, диагностика, лечение2221-996X2619-11562018-09-0118314014910.30895/2221-996X-2018-18-3-140-149132Genome-Editing and Biomedical Cell Products: Current State, Safety and EfficacyA. A. Goryaev0M. V. Savkina1K. M. Mefed2V. P. Bondarev3V. A. Merkulov4V. V. Tarasov5Scientific Centre for Expert Evaluation of Medicinal ProductsScientific Centre for Expert Evaluation of Medicinal ProductsScientific Centre for Expert Evaluation of Medicinal ProductsScientific Centre for Expert Evaluation of Medicinal ProductsScientific Centre for Expert Evaluation of Medicinal ProductsI. M. Sechenov First Moscow State Medical UniversityAdvances in ex vivo technologies of human genome editing have made it possible to develop new approaches to the treatment of genetic, oncological, infectious and other diseases, which may involve the use of biomedical cell products. However, despite the rapid development of these technologies and a large number of clinical trials conducted in many countries around the world, only 4 products (Strimvelis, Zalmoxis, Kymriah and Yescarta) containing ex vivo genetically modified human cells are authorised for use in the European Union and the United States of America. This paper considers three promising technologies (ZFN, TALEN and CRISPR) that allow for easy and effective editing of the genome at the sites of interest, thereby creating a platform for further development of the genetic engineering of human cells. It describes the technology of engineering chimeric antigen receptors (CARs). It also provides data on the efficacy and safety of the approved products: Strimvelis which contains autologous CD34+ cells transduced ex vivo with a retroviral vector containing adenosine deaminase gene, Zalmoxis which contains modified allogeneic T-cells, and two products: Kymriah and Yescarta which contain autologous T-cells with CARs to CD19 antigen, intended for the treatment of CD19+ hematological malignancies.https://www.biopreparations.ru/jour/article/view/169biomedical cell products (bmcp)cell therapygene therapychimeric antigen receptorgenome editingzfntalencrispr |
| spellingShingle | A. A. Goryaev M. V. Savkina K. M. Mefed V. P. Bondarev V. A. Merkulov V. V. Tarasov Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy Биопрепараты: Профилактика, диагностика, лечение biomedical cell products (bmcp) cell therapy gene therapy chimeric antigen receptor genome editing zfn talen crispr |
| title | Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy |
| title_full | Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy |
| title_fullStr | Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy |
| title_full_unstemmed | Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy |
| title_short | Genome-Editing and Biomedical Cell Products: Current State, Safety and Efficacy |
| title_sort | genome editing and biomedical cell products current state safety and efficacy |
| topic | biomedical cell products (bmcp) cell therapy gene therapy chimeric antigen receptor genome editing zfn talen crispr |
| url | https://www.biopreparations.ru/jour/article/view/169 |
| work_keys_str_mv | AT aagoryaev genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy AT mvsavkina genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy AT kmmefed genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy AT vpbondarev genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy AT vamerkulov genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy AT vvtarasov genomeeditingandbiomedicalcellproductscurrentstatesafetyandefficacy |