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

por 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.
Publicado 2021