Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus
After decades of investigation, gene therapy has received regulatory approval to treat hemophilia. However, since gene therapy investigations were initially conceived, other avenues of treatment have revolutionized the care of hemophilia. Emergent data is showing that gene therapy may not be as bene...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-11-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2023.1256919/full |
| _version_ | 1797629385884303360 |
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| author | John Puetz |
| author_facet | John Puetz |
| author_sort | John Puetz |
| collection | DOAJ |
| description | After decades of investigation, gene therapy has received regulatory approval to treat hemophilia. However, since gene therapy investigations were initially conceived, other avenues of treatment have revolutionized the care of hemophilia. Emergent data is showing that gene therapy may not be as beneficial as hoped and more toxic than planned. At a minimum, a reassessment of risk/benefit estimate of gene therapy for hemophilia is needed. |
| first_indexed | 2024-03-11T10:53:44Z |
| format | Article |
| id | doaj.art-09b7dc3f131147aaad0f7f4a5f159d82 |
| institution | Directory Open Access Journal |
| issn | 2296-858X |
| language | English |
| last_indexed | 2024-03-11T10:53:44Z |
| publishDate | 2023-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj.art-09b7dc3f131147aaad0f7f4a5f159d822023-11-13T11:53:52ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2023-11-011010.3389/fmed.2023.12569191256919Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virusJohn PuetzAfter decades of investigation, gene therapy has received regulatory approval to treat hemophilia. However, since gene therapy investigations were initially conceived, other avenues of treatment have revolutionized the care of hemophilia. Emergent data is showing that gene therapy may not be as beneficial as hoped and more toxic than planned. At a minimum, a reassessment of risk/benefit estimate of gene therapy for hemophilia is needed.https://www.frontiersin.org/articles/10.3389/fmed.2023.1256919/fullhemophiliagene therapyadeno-associated virusfactor VIIIfactor IXhepatocellular carcinoma |
| spellingShingle | John Puetz Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus Frontiers in Medicine hemophilia gene therapy adeno-associated virus factor VIII factor IX hepatocellular carcinoma |
| title | Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus |
| title_full | Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus |
| title_fullStr | Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus |
| title_full_unstemmed | Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus |
| title_short | Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus |
| title_sort | emergent data influences the risk benefit assessment of hemophilia gene therapy using recombinant adeno associated virus |
| topic | hemophilia gene therapy adeno-associated virus factor VIII factor IX hepatocellular carcinoma |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2023.1256919/full |
| work_keys_str_mv | AT johnpuetz emergentdatainfluencestheriskbenefitassessmentofhemophiliagenetherapyusingrecombinantadenoassociatedvirus |