Cholesterol metabolism in aging and age-related disorders

G Saher - Annual Review of Neuroscience, 2023 - annualreviews.org
All mammalian cell membranes contain cholesterol to maintain membrane integrity. The
transport of this hydrophobic lipid is mediated by lipoproteins. Cholesterol is especially …

Adeno-associated virus as a delivery vector for gene therapy of human diseases

JH Wang, DJ Gessler, W Zhan, TL Gallagher… - Signal Transduction and …, 2024 - nature.com
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 for liver diseases—progress and challenges

N Zabaleta, C Unzu, ND Weber… - Nature Reviews …, 2023 - nature.com
Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

CRISPR-Based Therapy for Hereditary Angioedema

DM Cohn, P Gurugama, M Magerl… - … England Journal of …, 2024 - Mass Medical Soc
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 …

Centriole signaling restricts hepatocyte ploidy to maintain liver integrity

VC Sladky, H Akbari, D Tapias-Gomez… - Genes & …, 2022 - genesdev.cshlp.org
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 …

[HTML][HTML] Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy

ND Weber, L Odriozola, I Ros-Gañán, G García-Porrero… - JHEP Reports, 2023 - Elsevier
Abstract Background & Aims Gene therapy using recombinant adeno-associated virus
(rAAV) vector carrying multidrug resistance protein 3 (MDR3) coding sequence (AAV8 …

The ploidy state as a determinant of hepatocyte proliferation

SR Wilson, AW Duncan - Seminars in liver disease, 2023 - thieme-connect.com
The liver's unique chromosomal variations, including polyploidy and aneuploidy, influence
hepatocyte identity and function. Among the most well-studied mammalian polyploid cells …

[HTML][HTML] Distinct hepatocyte identities in liver homeostasis and regeneration

F Chen, K Schönberger, JS Tchorz - JHEP Reports, 2023 - Elsevier
The process of metabolic liver zonation is spontaneously established by assigning
distributed tasks to hepatocytes along the porto-central blood flow. Hepatocytes fulfil critical …

Immuno-inflammatory in vitro hepatotoxicity models to assess side effects of biologicals exemplified by aldesleukin

LA Roser, S Luckhardt, N Ziegler, D Thomas… - Frontiers in …, 2023 - frontiersin.org
Introduction Hepatotoxicity induced by immunotherapeutics is an appearing cause for
immune-mediated drug-induced liver injury. Such immuno-toxic mechanisms are difficult to …

Binucleated human hepatocytes arise through late cytokinetic regression during endomitosis M phase

GS Darmasaputra, CC Geerlings… - Journal of Cell …, 2024 - rupress.org
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 …