K16ApoE-mediated delivery across the blood-brain barrier: mechanisms, applications, and translational challenges.
It is shown that co-administration of K16ApoE with therapeutic proteins can mediate high concentrations of this protein in brain tissue, which can yield significant substrate clearance and profound neurologic improvement and survival benefits in animal models of late-infantile neuronal ceroid lipofuscinosis (LINCL; CLN2 disease).