What question did this study set out to answer?

Investigate immune responses to the adeno-associated virus vector and the factor IX transgene product in individuals receiving liver-directed gene transfer for hemophilia B.

May 1, 2004Molecular TherapyOpen Access

Immune Responses to AAV and to Factor IX in a Phase I Study of AAV-Mediated, Liver-Directed Gene Transfer for Hemophilia B

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Implication

Phase I trial reports immune responses to AAV and factor IX in hemophilia B, highlighting immunological barriers to liver-directed gene transfer.

Key Points

Investigate immune responses to the adeno-associated virus vector and the factor IX transgene product in individuals receiving liver-directed gene transfer for hemophilia B.
Conducted a Phase I clinical trial assessing liver-directed adeno-associated virus (AAV) vector-mediated gene transfer encoding human factor IX.
Evaluated host immune responses directed against the viral vector capsid and the expressed factor IX protein following hepatic delivery.
Characterized the immunological factors influencing vector persistence and sustained transgene expression in treated patients.

Cite This Study

A 2004 study studied this question.

synapsesocial.com/papers/6a7004bdc92390ac2d07b291 https://doi.org/10.1016/j.ymthe.2004.06.940

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Implication

Phase I trial reports immune responses to AAV and factor IX in hemophilia B, highlighting immunological barriers to liver-directed gene transfer.

Immune Responses to AAV and to Factor IX in a Phase I Study of AAV-Mediated, Liver-Directed Gene Transfer for Hemophilia B

Discussion

Member takes

Implication

Key Points

Cite This Study

Immune Responses to AAV and to Factor IX in a Phase I Study of AAV-Mediated, Liver-Directed Gene Transfer for Hemophilia B

Discussion

Member takes

Implication

Key Points

Cite This Study

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