Asociación Española para el Estudio del Hígado

🧬 A major milestone in gene therapy for liver disease! For the first time, researchers have published a peer-reviewed clinical case demonstrating successful in vivo base editing in a patient with alpha-1 antitrypsin deficiency. 🔬 Published in the @JHepatology, the study reports: 💠First peer-reviewed publication of a successfully treated PiZZ AATD patient with in vivo gene therapy 💠High correction efficiency, precise editing, and promising safety profile 💠Conversion of homozygous to heterozygous genotype – a major therapeutic achievement 💠Potential to treat numerous hereditary liver diseases and rare variants These promising findings mark an important step towards precise, minimally invasive treatments for inherited liver diseases. While further research is needed to establish long-term safety and efficacy, the study highlights the transformative potential of gene editing in hepatology. 🔗 Read the press release: easl.eu/gene-correction-ther… 👉Read the article: journal-of-hepatology.eu/art… @DebbieShawcros1 @analleo #MHH