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Description
To assess the safety of systemic administration of a novel self complementary AAV vector (scAAV2/8-LP1-hFIXco) in adults with severe hemophilia B at up to three dose levels. We predict that the systemic administration of scAAV2/8-LP1-hFIXco vector pseudotyped with AAV8 capsid protein and encoding the human FIX (hFIX) gene will be safe with absence of persistent Grade III or greater dose limiting toxicity. Any Grade I-II toxicity is likely to be dose dependent and reversible. To test this hypothesis we have developed a comprehensive clinical monitoring plan that will detect and document any adverse events following gene transfer.
Status | Finished |
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Effective start/end date | 10/1/13 → 1/31/16 |
Funding
- St Jude Childrens Research Hospital
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Projects
- 1 Finished
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AGT4HB - Viral Vector for Gene Transfer in Subjects with Hemophilia B
Radulescu, V. (PI)
St Jude Childrens Research Hospital
10/1/13 → 1/31/16
Project: Research project