Gene therapy to the blood ‐brain barrier with resulting protein secretion as a strategy for treatment of Niemann Picks type C2 disease

In conclusion, non‐viral gene therapy to rBCECs leads to protein secretion and signifies a method for NPC2 to target cells inside the CNS otherwise inaccessible due to the pres ence of the BBB. However, obtaining high transfection efficiencies is crucial in order to achieve sufficient therapeutic effects.

Source: Journal of Neurochemistry - February 20, 2020 Category: Neuroscience Authors: Eva Hede, Christine Bodelund Christiansen, Christian W ürtz Heegaard, Torben Moos, Annette Burkhart Tags: ORIGINAL ARTICLE Source Type: research