Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells.
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R. D'Hooge | R. Lüllmann-Rauch | D. Hartmann | V. Gieselmann | P. De Deyn | U. Matzner | F. Schestag