Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia
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| Format: | Article |
| Language: | English |
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Elsevier
2025-06-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050125000762 |
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| _version_ | 1850040088225906688 |
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| author | Stefano Rivella |
| author_facet | Stefano Rivella |
| author_sort | Stefano Rivella |
| collection | DOAJ |
| format | Article |
| id | doaj-art-d2ecd1454d454b37980fe73c1bb4ff8a |
| institution | DOAJ |
| issn | 2329-0501 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj-art-d2ecd1454d454b37980fe73c1bb4ff8a2025-08-20T02:56:09ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012025-06-0133210148110.1016/j.omtm.2025.101481Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemiaStefano Rivella0Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA; University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA; Raymond G. Perelman Center for Cellular and Molecular Therapeutics-The Children’s Hospital of Philadelphia, Philadelphia, PA, USA; Penn Center for Musculoskeletal Disorders, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA; Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia, PA, USA; Corresponding author: Stefano Rivella, Ph.D., Children’s Hospital of Philadelphia, Abramson Research Center, 3615 Civic Center Blvd, Room 316B, Philadelphia, PA 19104, USA.http://www.sciencedirect.com/science/article/pii/S2329050125000762 |
| spellingShingle | Stefano Rivella Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia Molecular Therapy: Methods & Clinical Development |
| title | Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia |
| title_full | Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia |
| title_fullStr | Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia |
| title_full_unstemmed | Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia |
| title_short | Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia |
| title_sort | assessing the safety of gene therapy vectors expressing an enhanced gamma globin gene for the cure of sickle cell anemia |
| url | http://www.sciencedirect.com/science/article/pii/S2329050125000762 |
| work_keys_str_mv | AT stefanorivella assessingthesafetyofgenetherapyvectorsexpressinganenhancedgammaglobingeneforthecureofsicklecellanemia |