Growth Advantage of Corrected Hepatocytes in a Juvenile Model of Methylmalonic Acidemia Following Liver Directed Adeno-Associated Viral Mediated Nuclease-Free Genome Editing
MOLECULAR GENETICS AND METABOLISM（2022）
Key words
Genome editing,Gene therapy,Methylmalonic acidemia,MMA,Fibroblast growth factor 21,FGF21,Organic acidemia,Adeno-associated virus,AAV
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