The New England journal of medicine, 2022-07, Vol.387 (3), p.237-247
2022
Volltextzugriff (PDF)
Details
- Autor(en) / Beteiligte
- Titel
- Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B
- Ist Teil von
- The New England journal of medicine, 2022-07, Vol.387 (3), p.237-247
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2022
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- Nine of 10 patients with hemophilia B who received a liver-directed adeno-associated viral vector containing a hyperactive variant of factor IX maintained factor IX activity for up to 42 months. Thrombosis developed in 1 patient.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa2119913Titel-ID: cdi_proquest_journals_2692175909
- Format
- –
- Schlagworte
- Adverse events, Antibodies, Body weight, Clinical trials, Coagulation, Coagulation factors, Dependovirus - genetics, Design, Disease prevention, Factor IX - analysis, Factor IX - genetics, Factor IX deficiency, Follow-Up Studies, Gene therapy, Genetic Therapy - adverse effects, Genetic Therapy - methods, Genetic Vectors - therapeutic use, Genetics, Genetics General, Genomes, Glucocorticoids, Glucocorticoids - adverse effects, Glucocorticoids - therapeutic use, Hematology, Hemophilia, Hemophilia B - genetics, Hemophilia B - metabolism, Hemophilia B - therapy, Humans, Immune response, Immunosuppression, Immunosuppressive Agents - adverse effects, Immunosuppressive Agents - therapeutic use, Liver, Mutation, Oncology, Oncology General, Patients, Pediatrics, Pediatrics General, Prophylaxis, Statistical analysis, Tacrolimus, Tacrolimus - adverse effects, Tacrolimus - therapeutic use, Thrombosis, Transaminases - analysis