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Base Editing Reduces Misfolded Protein Accumulation and Toxicity in Alpha-1 Antitrypsin Deficient Patient iPSC-Hepatocytes
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Adenine base editing reduces misfolded protein accumulation and toxicity in alpha-1 antitrypsin deficient patient iPSC-hepatocytes
R. Werder, J. Kaserman, M. Packer, et al. (2021). Molecular Therapy. Cited 24 times.
https://doi.org/10.1016/j.ymthe.2021.06.021
