Adenine base editing reduces misfolded protein accumulation and toxicity in alpha-1 antitrypsin deficient patient iPSC-hepatocytes

Alpha-1 antitrypsin deficiency (AATD) is most commonly caused by the Z mutation, a single-base substitution that leads to AAT protein misfolding and associated liver and lung disease. In this study, we apply adenine base editors to correct the Z mutation in patient induced pluripotent stem cells (iP...

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Detalles Bibliográficos
Autores principales: Werder, Rhiannon B., Kaserman, Joseph E., Packer, Michael S., Lindstrom-Vautrin, Jonathan, Villacorta-Martin, Carlos, Young, Lauren E., Aratyn-Schaus, Yvonne, Gregoire, Francine, Wilson, Andrew A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571173/
https://www.ncbi.nlm.nih.gov/pubmed/34217893
http://dx.doi.org/10.1016/j.ymthe.2021.06.021

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