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Article

Gene therapy for hemophilia B in immature mice using ScAAV-DJ/8-LP1-hFIXco vector

2023-10-13

Abstract excerpt

<title>Abstract</title> <p>Hemophilia B is an X-linked bleeding disorder that derives from the mutation in the gene encoding coagulation factor IX(FIX). Gene therapy provided the promising hope to cure the disease. Currently, pure virus injection is exclusively limited to adults, resulting in favorable outcomes. However, this approach has rarely been tested in pediatric patients due to its considerable impact on...

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Literature Corpus work
2d4c1803-43cd-5c3a-93c1-41e841c3310b
DOI
10.21203/rs.3.rs-3379986/v1
Open publication

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Gene therapy for hemophilia B in immature mice using ScAAV-DJ/8-LP1-hFIXco vectorDOI 10.21203/rs.3.rs-3379986/v1
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