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Astrocyte-targeted gene delivery of interleukin 2 specifically increases brain-...

Yshii, L; Pasciuto, E; Bielefeld, P; Mascali, L; Lemaitre, P; Marino, M; Dooley, J; Kouser, L; Verschoren, S; Lagou, V; Kemps, H; Gervois, P; Boer, A

The ability of immune-modulating biologics to prevent and reverse pathology has transformed recent clinical practice. Full utility in the neuroinflammation space, however, requires identification of both effective targets for local immune modulation and a delivery system capable of crossing the blood-brain barrier. The recent identification and characterization of a small population of regulatory T (Treg) cells...


AAV-mediated delivery of an anti-BACE1 VHH alleviates pathology in an Alzheimer...

Marino, M; Zhou, L; Rincon, MY; Callaerts-Vegh, Z; Verhaert, J; Wahis, J; Creemers, E; Yshii, L; Wierda, K; Saito, T; Marneffe, C; Voytyuk, I

Single domain antibodies (VHHs) are potentially disruptive therapeutics, with important biological value for treatment of several diseases, including neurological disorders. However, VHHs have not been widely used in the central nervous system (CNS), largely because of their restricted blood-brain barrier (BBB) penetration. Here, we propose a gene transfer strategy based on BBB-crossing adeno-associated virus (...


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