Lipid Nanoparticle-Mediated Liver-Specific Gene Therapy for Hemophilia B

• Main Authors: Brijesh Lohchania (Author), Porkizhi Arjunan (Author), Gokulnath Mahalingam (Author), Abinaya Dandapani (Author), Pankaj Taneja (Author), Srujan Marepally (Author)
• Format: Bog
• Udgivet: MDPI AG, 2024-11-01T00:00:00Z.
• Fag: article
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<b>Background</b>/<b>Objectives</b>: Hemophilia B is a hereditary bleeding disorder due to the production of liver malfunctional factor IX (FIX). Gene therapy with viral vectors offers a cure. However, applications are limited due to pre-existing antibodies, eligibility for c...