Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy-Reference-Cited by-同舟云学术

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Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy

Published:2018-01 Issue:1 Volume:26 Page:14-16
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

VandenDriessche Thierry,Chuah Marinee K.

Funder

Fonds voor Wetenschappelijk Onderzoek, Vrije Universiteit Brussel – Industrieel Onderzoeksfonds

Strategisch Onderzoeksproject – Groeier Program

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference17 articles.

1. Hemophilia Gene Therapy: Ready for Prime Time?;VandenDriessche;Hum. Gene Ther.,2017

2. Cellular and genetic therapies for haemophilia;Lillicrap;Haemophilia,2006

3. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N. Engl. J. Med.,2014

4. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant;George;N. Engl. J. Med.,2017

5. X-linked thrombophilia with a mutant factor IX (factor IX Padua);Simioni;N. Engl. J. Med.,2009

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1. Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy;Current Gene Therapy;2024-08

2. Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B;Expert Review of Hematology;2023-11-12

3. An AsCas12f-based compact genome-editing tool derived by deep mutational scanning and structural analysis;Cell;2023-10

4. Gene Therapy Approaches for the Treatment of Hemophilia B;International Journal of Molecular Sciences;2023-06-28

5. Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response;Nature Communications;2022-11-25

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