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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

by Li, Mingjin , Wong, Wing Y , Pasi, K. John , Symington, Emily , Rangarajan, Savita , Russell, Chris B , Pierce, Glenn F , Madan, Bella , Laffan, Michael , Lester, Will , Mitchell, Nina

in Bleeding / Body weight / Coagulation factors / Deoxyribonucleic acid / DNA / Enzymes / Factor VIII deficiency / Gene therapy / Genomes / Hemophilia / Pharmacodynamics / Safety

2020

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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

by Li, Mingjin , Wong, Wing Y , Pasi, K. John , Symington, Emily , Rangarajan, Savita , Russell, Chris B , Pierce, Glenn F , Madan, Bella , Laffan, Michael , Lester, Will , Mitchell, Nina

in Bleeding / Body weight / Coagulation factors / Deoxyribonucleic acid / DNA / Enzymes / Factor VIII deficiency / Gene therapy / Genomes / Hemophilia / Pharmacodynamics / Safety

2020

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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

by Li, Mingjin , Wong, Wing Y , Pasi, K. John , Symington, Emily , Rangarajan, Savita , Russell, Chris B , Pierce, Glenn F , Madan, Bella , Laffan, Michael , Lester, Will , Mitchell, Nina

in Bleeding / Body weight / Coagulation factors / Deoxyribonucleic acid / DNA / Enzymes / Factor VIII deficiency / Gene therapy / Genomes / Hemophilia / Pharmacodynamics / Safety

2020

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Journal Article

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

Li, Mingjin,

Wong, Wing Y,

Pasi, K. John,

Symington, Emily,

Rangarajan, Savita,

Russell, Chris B,

Pierce, Glenn F,

Madan, Bella,

Laffan, Michael,

Lester, Will,

Mitchell, Nina

2020

Overview

In 15 participants with hemophilia A treated with a single infusion of AAV5 vector containing the factor VIII gene, 7 who received 6×10 13 vector genomes per kilogram had a median factor VIII level of 20 IU per deciliter, and 6 who received 4×10 13 vg per kilogram had a median level of 13 IU per deciliter. Factor VIII levels decreased in years 2 and 3 of follow-up, but the median annualized rate of bleeding events among these 13 participants remained zero.

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Publisher

Massachusetts Medical Society

Subject

Bleeding

/ Body weight

/ Coagulation factors

/ Deoxyribonucleic acid

/ DNA

/ Enzymes

/ Factor VIII deficiency