Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer
Nathwani A, Reiss U, Tuddenham E, Chowdary P, McIntosh J, Riddell A, Pie J, Mahlangu J, Recht M, Shen Y, Halka K, Meagher M, Nienhuis A, Davidoff A, Mangles S, Morton C, Junfang Z, Radulescu V. Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer. Blood 2018, 132: 491. DOI: 10.1182/blood-2018-99-118334.Peer-Reviewed Original ResearchEntity's Board of DirectorsSelf-complementary adeno-associated virusVector preparationsFollow-upSevere HBDose cohortsHB patientsAAV-mediated transgene expressionEfficacy of gene transferGene transferLiver enzymesYear followHemophilia B (HBTransgene expressionLoss of transgene expressionFIX activity levelsPlasma FIX levelsSerotype 8 capsidAnnualized bleeding rateElevation of serum alanine aminotransferaseMedian Follow-UpAdeno-associated virusIncidence of hepatotoxicityB (HBRisk of hepatotoxicity
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply