Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A-Reference-Cited by-同舟云学术

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Published:2022-03 Issue: Volume:24 Page:20-29
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Elkouby Liron,Armour Sean M.^ORCID,Toso Raffaella^ORCID,DiPietro Marti,Davidson Robert J.,Nguyen Giang N.^ORCID,Willet Mallory^ORCID,Kutza Stephanie,Silverberg Joseph,Frick Jennifer,Crosariol Marco,Wang Yuhuan,Wang Chuansong,High Katherine A.,Sabatino Denise E.^ORCID,Anguela Xavier M.^ORCID

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference41 articles.

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

2. Hemophilia B gene therapy with a high-specific-activity factor IX variant;George;N. Engl. J. Med.,2017

3. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B;Miesbach;Blood,2018

4. Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region;Hoeben;Blood,1995

5. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production;Lynch;Hum. Gene Ther.,1993

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1. Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice;Nature Communications;2024-08-21

2. Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques;Molecular Therapy;2024-07

3. Coagulation factor VIII regulates von Willebrand factor homeostasis in vivo;Journal of Thrombosis and Haemostasis;2023-12

4. Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy;Biology Open;2023-09-15

5. Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice;Journal of Thrombosis and Haemostasis;2023-08