Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy-Reference-Cited by-同舟云学术

Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy

Published:2023-09 Issue: Volume:30 Page:502-514
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Kashiwakura Yuji,Endo Kazuhiro,Ugajin Atsushi,Kikuchi Tomohiro,Hishikawa Shuji,Nakamura Hitoyasu,Katakai Yuko,Baatartsogt Nemekhbayar,Hiramoto Takafumi,Hayakawa Morisada,Kamoshita Nobuhiko,Yamazaki Shoji,Kume Akihiro,Mori Harushi,Sata Naohiro,Sakata Yoichi,Muramatsu Shin-ichi,Ohmori Tsukasa^ORCID

Funder

Japan Agency for Medical Research and Development

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference37 articles.

1. Hemophilia gene therapy: ushering in a new treatment paradigm?;George;Hematology. Am. Soc. Hematol. Educ. Program,2021

2. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B;Nathwani;N. Engl. J. Med.,2011

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

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

5. Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B;Chowdary;N. Engl. J. Med.,2022

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