- 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. Matzner, U., Schestag, F., Hartmann, D., Lüllmann-Rauch, R., D'Hooge, R., De Deyn, P.P., Gieselmann, V. Hum. Gene Ther. (2001)