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#gene editing Case report Open access Nov 2026

Liver biopsy confirms precise and efficient correction of SERPINA1 after in vivo Base Editing in a Patient with Alpha-1 Antitrypsin Deficiency.

BACKGROUND Alpha-1 antitrypsin deficiency (AATD) caused by the PI*ZZ genotype (Glu342Lys) is characterized by hepatic accumulation of misfolded AAT-Z protein and reduced circulating AAT levels, leading to progressive liver disease and pulmonary emphysema. Gene correction therapy represents a potentially curative approa...

S. Krooss, Tai-Hua Yang, Q. Yuan et al. · 0 citations
Open access Aug 2026

Hyperglycemia in Diabetes Drives Hepatic Fibrosis Through the Non-Enzymatic Function of HK2 and AGEs-mediated Matrix Stiffening

Diabetes is implicated in accelerating the progression of hepatic fibrosis, and empagliflozin (an SGLT2 inhibitor) has potential therapeutic effects on diabetic hepatic fibrosis. However, their underlying mechanisms are still largely uncharacterized. In this study, we found that diabetes obviously promoted hepatic fibr...

Chu-Xin Huang, Jia-Li Qian, Li Zhang et al. · 0 citations

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