Issue Cover (February 2021)

Abstract

Journal of NeurochemistryVolume 156, Issue 3 Issue CoverFree Access Issue Cover (February 2021) First published: 18 February 2021 https://doi.org/10.1111/jnc.15050 Read the full article: ‘Gene therapy to the blood–brain barrier with resulting protein secretion as a strategy for treatment of Niemann Picks type C2 disease’ by E. Hede, C. B. Christiansen, C. W. Heegaard, T. Moos, A. Burkhart, (J. Neurochem. 2021, vol. 156 (3), pp. 290–308) on doi:10.1111/jnc.14982 AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinked InRedditWechat Abstract Front cover: Gene therapy at the blood-brain barrier (BBB) serves as a promising strategy to treat the cerebral impact of Niemann Picks disease type C2. Genetically modifying BCECs cultured in an in vitro BBB model with astrocytes with genetic material encoding Niemann Picks type C2 protein (NPC2) results in a bidirectional secretion of recombinant NPC2. NPC2 deficient fibroblasts show high cholesterol deposits in their lysosomes, due to a failed transport of cholesterol within the cells. Recombinant NPC2 secreted from genetically modified BCECs can be taken up by the NPC2 deficient fibroblasts to restore the cholesterol transport within these cells. However, increasing the number of genetically modified cells and thereby the concentration of secreted NPC2, is crucial for obtaining a sufficient biological effect on the NPC2 deficient fibroblasts. Image Content: The image depicts NPC2 deficient fibroblasts stained for Vimentin. Scalebar 20µm Read the full article ‘Gene therapy to the blood–brain barrier with resulting protein secretion as a strategy for treatment of Niemann Picks type C2 disease’ by E. Hede, C. B. Christiansen, C. W. Heegaard, T. Moos, A. Burkhart, (J. Neurochem. 2021, vol. 156 (3), pp. 290–308) on doi:10.1111/jnc.14982 Volume156, Issue3February 2021 RelatedInformation