An isogenic panel of App knock-in mouse models: Profiling β-secretase inhibition and endosomal abnormalities

Sci Adv. 2022 Jun 10;8(23):eabm6155. doi: 10.1126/sciadv.abm6155. Epub 2022 Jun 8.

Abstract

We previously developed single App knock-in mouse models of Alzheimer's disease (AD) that harbor the Swedish and Beyreuther/Iberian mutations with or without the Arctic mutation (AppNL-G-F and AppNL-F mice). We have now generated App knock-in mice devoid of the Swedish mutations (AppG-F mice) and evaluated its characteristics. Amyloid β peptide (Aβ) pathology was exhibited by AppG-F mice from 6 to 8 months of age and was accompanied by neuroinflammation. Aβ-secretase inhibitor, verubecestat, attenuated Aβ production in AppG-F mice, but not in AppNL-G-F mice, indicating that the AppG-F mice are more suitable for preclinical studies of β-secretase inhibition given that most patients with AD do not carry the Swedish mutations. Comparison of isogenic App knock-in lines revealed that multiple factors, including elevated C-terminal fragment β (CTF-β) and humanization of Aβ might influence endosomal alterations in vivo. Thus, experimental comparisons between different isogenic App, knock-in mouse lines will provide previously unidentified insights into our understanding of the etiology of AD.

MeSH terms

  • Alzheimer Disease* / genetics
  • Alzheimer Disease* / pathology
  • Amyloid Precursor Protein Secretases / genetics
  • Amyloid beta-Peptides / genetics
  • Animals
  • Disease Models, Animal*
  • Gene Knock-In Techniques
  • Humans
  • Mice
  • Mice, Transgenic

Substances

  • Amyloid beta-Peptides
  • Amyloid Precursor Protein Secretases