Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

All mammalian cell membranes contain cholesterol to maintain membrane integrity. The
transport of this hydrophobic lipid is mediated by lipoproteins. Cholesterol is especially …

Background Hereditary angioedema is a rare genetic disease characterized by severe and
unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy that is based …

Binucleated polyploid cells are common in many animal tissues, where they arise by
endomitosis, a non-canonical cell cycle in which cells enter M phase but do not undergo …

Cell-free DNA (cfDNA) tests use small amounts of DNA in the bloodstream as biomarkers.
While it is thought that cfDNA is largely released by dying cells, the proportion of dying cells' …

Metabolic dysfunction‐associated steatohepatitis (MASH) is a leading cause of chronic liver
disease with few therapeutic options. To narrow the translational gap in the development of …

The process of metabolic liver zonation is spontaneously established by assigning
distributed tasks to hepatocytes along the porto-central blood flow. Hepatocytes fulfil critical …

ABSTRACT Introduction Congenital hemophilia B (HB) is an X-linked bleeding disorder
resulting in Factor IX (FIX) deficiency and bleeding of variable severity. There is no cure for …

Hepatocyte polyploidization is a tightly controlled process that is initiated at weaning and
increases with age. The proliferation of polyploid hepatocytes in vivo is restricted by the …