Abstract
In this issue of Blood, Nair et al demonstrate that AAV-directed gene therapy using a new bioengineered FIX transgene provides higher FIX activity and superior hemostatic efficacy than other FIX variants and may allow for lower and potentially safer vector doses in future human clinical trials.
MeSH terms
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Factor IX / genetics
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Genetic Therapy
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Hemophilia B* / genetics
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Hemophilia B* / therapy
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Humans