Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.

"Declaration of interests A.T.v.d.P. has received consulting fees from Sanofi Genzyme and has provided consulting services, participated in advisory board meetings and received grants for premarketing studies and research from industries via agreements between Erasmus MC and the industry. W.W.M.P.P. and A.T.v.d,P. are advisors of LentiCure B.V., a company for the development of lentiviral gene therapy. F.C., E.C.V., A.T.v.d.P., and W.W.M.P.P. are inventors on patents in the field of lentiviral gene therapy."

"This work was supported by the "Finding a cure for Hunter Disease" Foundation, the Sophia Foundation (grant S18-59), Metakids (grants 2018-083 and 2021-094), the 10.13039/501100004243Prinses Beatrix Spierfonds (grant W.OP20-04), the Hersenstichting (grant DR-2021-00386), 10.13039/501100010573Dioraphte (project 20210367), and the Dutch Research Agenda (NWA) grant “Cure4Life” from the 10.13039/501100003246Dutch Research Council (NWO). We thank Prof. Dr. Brian Bigger for kindly providing the transgenes IDSco and IDS.ApoE2co, and Hélène Gleitz for the technical advice. Fabio Catalano has been granted a license to use BioRender for the making of the graphical abstract (agreement no. KY25WFNHYP; created with biorender.com)."