Article

Human Umbilical Cord Mesenchymal Stem Cell-Based Gene Therapy for Hemophilia B using scAAV-DJ/8-LP1-hFIXco Transduction

2024-02-27

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold> Hemophilia B is an X-linked bleeding disorder caused by a mutation in the gene responsible for encoding coagulation factor IX (FIX). Gene therapy offers promising potential for curing this disease. However, the current method of relatively high dosage of virus injection carries inherent risks. The purpose of this study was to introduce a novel scAAV-DJ/8-LP1-hFIX...

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Literature Corpus work: 2311e05b-ba37-52a8-9789-de0dcb197f8e
DOI: 10.21203/rs.3.rs-3950368/v1

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