Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A-Reference-Cited by-同舟云学术

Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

Published:2020-06 Issue: Volume:17 Page:465-477
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

El-Akabawy Nadia,Rodriguez Martin,Ramamurthy Ritu,Rabah Andrew,Trevisan Brady,Morsi Alshaimaa,George Sunil,Shields Jordan,Meares Diane,Farland Andrew,Atala Anthony,Doering Christopher B.,Spencer H. Trent,Porada Christopher D.,Almeida-Porada Graça

Funder

NIH

NHLBI

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference74 articles.

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3. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies;Shi;J. Clin. Invest.,2006

4. Phenotypic correction of hemophilia A by postnatal intraperitoneal transplantation of FVIII-expressing MSC;Porada;Blood,2010

5. AAV5-factor VIII gene transfer in severe hemophilia A;Rangarajan;N. Engl. J. Med.,2017

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1. Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity;Nature Communications;2023-07-14

2. Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII;Frontiers in Immunology;2022-12-15

3. Autologous bone marrow-derived MSCs engineered to express oFVIII-FLAG engraft in adult sheep and produce an effective increase in plasma FVIII levels;Frontiers in Immunology;2022-12-02

4. Lentiviral Vectors for Gene Therapy;eLS;2022-09-27

5. In Vitro Conditioning of Adipose-Derived Mesenchymal Stem Cells by the Endothelial Microenvironment: Modeling Cell Responsiveness towards Non-Genetic Correction of Haemophilia A;International Journal of Molecular Sciences;2022-06-30