Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

BACKGROUND AND AIMS: Adeno‐associated viral (AAV) gene therapy has shown great promise as an alternative treatment for metabolic disorders managed using liver transplantation, but remains limited by transgene loss and genotoxicity. Our study aims to test an AAV vector with a promoterless integrating...

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Detalles Bibliográficos
Autores principales: Chandler, Randy J., Venturoni, Leah E., Liao, Jing, Hubbard, Brandon T., Schneller, Jessica L., Hoffmann, Victoria, Gordo, Susana, Zang, Shengwen, Ko, Chih‐Wei, Chau, Nelson, Chiang, Kyle, Kay, Mark A., Barzel, Adi, Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8252383/
https://www.ncbi.nlm.nih.gov/pubmed/32976669
http://dx.doi.org/10.1002/hep.31570

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8252383/
https://www.ncbi.nlm.nih.gov/pubmed/32976669
http://dx.doi.org/10.1002/hep.31570

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