Bringing Gene Therapy Into Real World Clinical Practice
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach +2 more
wiley +1 more source
Link between Metabolic Dysfunction-Associated Steatotic Liver Disease and Cardiovascular Diseases. [PDF]
Shen D, Chen R, Ye S.
europepmc +1 more source
Italian Patients Journey for Gene Therapy in Haemophilia A
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno +19 more
wiley +1 more source
Metabolic Dysfunction-Associated Steatotic Liver Disease: Clinical Heterogeneity, Risk Stratification, and Personalized Care. [PDF]
Vo TD.
europepmc +1 more source
ABSTRACT Aim Steatotic liver disease (SLD) encompasses a heterogeneous spectrum with varying risks of hepatocellular carcinoma (HCC). Limited sample sizes limit the development of predictive models, particularly for rare outcomes. This study evaluated whether generative artificial intelligence (AI)‐based synthetic data augmentation can enhance HCC risk
Masaya Sato +12 more
wiley +1 more source
Editorial: Liver and Non-Liver Outcomes Between Different Subtypes of Steatotic Liver Disease. [PDF]
Huang JF, Yu ML, Chuang WL.
europepmc +1 more source
We carried out proteomic analysis of serum extracellular vesicles to identify biomarkers predictive of hepatocellular carcinoma (HCC) development in patients with chronic hepatitis B receiving nucleos(t)ide analog therapy. Candidate proteins identified through proteomic analysis were subsequently quantified in serum by ELISA.
Emi Sometani +21 more
wiley +1 more source
Conducting the Orchestra: Nonparenchymal Cells Direct Hepatocyte Functions in Steatotic Liver Disease. [PDF]
Jaeger JW, Schwabe RF.
europepmc +1 more source
Pemafibrate Improves Cholestatic Markers Regardless of the Presence of Primary Biliary Cholangitis
Pemafibrate improves liver function markers related to bile flow in primary biliary cholangitis (PBC), but whether this effect is disease‐specific remains unclear. In this study, pemafibrate reduced these markers in patients with and without PBC, suggesting broader effects on bile flow beyond PBC.
Ryohei Tanigawa +5 more
wiley +1 more source
Helicobacter pylori Infection and Metabolic Dysfunction-Associated Steatotic Liver Disease: A Prospective Study and Meta-Analysis. [PDF]
Zhao L +16 more
europepmc +1 more source

