Therapeutic Genome Editing with CRISPR/Cas9 in a Humanized Mouse Model Ameliorates Α1-Antitrypsin Deficiency Phenotype
Research Perspectives of Microbiology and Biotechnology Vol 9（2025）
Key words
alpha 1-antitrypsin deficiency,CRISPR/Cas9 gene editing,Liver fibrosis,Protein aggregation
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