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

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

<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...

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Bibliographic Details
Main Authors: Brijesh Lohchania, Porkizhi Arjunan, Gokulnath Mahalingam, Abinaya Dandapani, Pankaj Taneja, Srujan Marepally
Format: Article
Language:English
Published: MDPI AG 2024-11-01
Series:Pharmaceutics
Subjects:
non-viral delivery
FIX-L (Padua mutant)
hemophilia B
lipid nanoparticles
gene therapy
Online Access:https://www.mdpi.com/1999-4923/16/11/1427
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