Current State of Human Gene Therapy: Approved Products and Vectors
In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-10-01
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| Series: | Pharmaceuticals |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1424-8247/16/10/1416 |
| _version_ | 1797572643052847104 |
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| author | Aladdin Y. Shchaslyvyi Svitlana V. Antonenko Maksym G. Tesliuk Gennadiy D. Telegeev |
| author_facet | Aladdin Y. Shchaslyvyi Svitlana V. Antonenko Maksym G. Tesliuk Gennadiy D. Telegeev |
| author_sort | Aladdin Y. Shchaslyvyi |
| collection | DOAJ |
| description | In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments, are now being addressed at their root cause—the genetic level. Presently, the gene therapy landscape stands adorned with 22 approved in vivo and ex vivo products, including IMLYGIC, LUXTURNA, Zolgensma, Spinraza, Patisiran, and many more. In this comprehensive exploration, we delve into a rich assortment of 16 drugs, from siRNA, miRNA, and CRISPR/Cas9 to DNA aptamers and TRAIL/APO2L, as well as 46 carriers, from AAV, AdV, LNPs, and exosomes to naked mRNA, sonoporation, and magnetofection. The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challenges faced in the practical use of gene therapy and its future potential. |
| first_indexed | 2024-03-10T20:58:54Z |
| format | Article |
| id | doaj.art-9b3b571dc8944b3caf80b6d3c3abe26a |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-10T20:58:54Z |
| publishDate | 2023-10-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-9b3b571dc8944b3caf80b6d3c3abe26a2023-11-19T17:42:11ZengMDPI AGPharmaceuticals1424-82472023-10-011610141610.3390/ph16101416Current State of Human Gene Therapy: Approved Products and VectorsAladdin Y. Shchaslyvyi0Svitlana V. Antonenko1Maksym G. Tesliuk2Gennadiy D. Telegeev3Institute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, UkraineInstitute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, UkraineInstitute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, UkraineInstitute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, UkraineIn the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments, are now being addressed at their root cause—the genetic level. Presently, the gene therapy landscape stands adorned with 22 approved in vivo and ex vivo products, including IMLYGIC, LUXTURNA, Zolgensma, Spinraza, Patisiran, and many more. In this comprehensive exploration, we delve into a rich assortment of 16 drugs, from siRNA, miRNA, and CRISPR/Cas9 to DNA aptamers and TRAIL/APO2L, as well as 46 carriers, from AAV, AdV, LNPs, and exosomes to naked mRNA, sonoporation, and magnetofection. The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challenges faced in the practical use of gene therapy and its future potential.https://www.mdpi.com/1424-8247/16/10/1416human gene therapygene therapy drugsviral vectorsnon-viral vectorsphysical delivery methodshuman gene therapy products |
| spellingShingle | Aladdin Y. Shchaslyvyi Svitlana V. Antonenko Maksym G. Tesliuk Gennadiy D. Telegeev Current State of Human Gene Therapy: Approved Products and Vectors Pharmaceuticals human gene therapy gene therapy drugs viral vectors non-viral vectors physical delivery methods human gene therapy products |
| title | Current State of Human Gene Therapy: Approved Products and Vectors |
| title_full | Current State of Human Gene Therapy: Approved Products and Vectors |
| title_fullStr | Current State of Human Gene Therapy: Approved Products and Vectors |
| title_full_unstemmed | Current State of Human Gene Therapy: Approved Products and Vectors |
| title_short | Current State of Human Gene Therapy: Approved Products and Vectors |
| title_sort | current state of human gene therapy approved products and vectors |
| topic | human gene therapy gene therapy drugs viral vectors non-viral vectors physical delivery methods human gene therapy products |
| url | https://www.mdpi.com/1424-8247/16/10/1416 |
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