Issue Cover (February 2021)

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 NPC2deficient fibroblasts. Image Content: The image depicts NPC2 deficient fibroblasts stained for Vimentin. Scalebar 20µmRead 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