Sustained Long-Term Disease Correction in a Murine Model of MPSII Following Stem Cell Gene Therapy

The authors observed sustained IDS enzyme activity in organs of long-term IDS. ApoEII-treated MPSII mice, similar to those analyzed six months post-treatment, with continued clearance of storage material in the brain and peripheral organs, maintained correction of astrogliosis, microgliosis and correction of altered cytokines and chemokines.