NOD/SCID/MPSVII mice that received transplants of human CD34⁺ cells showed correction of tissue pathology in some engrafted tissues.

The tissues of NOD/SCID/MPSVII mice that were either untreated or received transplants of human CD34⁺ cells were stained with toluidine blue. Lysosomes distended with storage material appear as clear, foamy areas by this method. In the bone marrow of NOD/SCID/MPSVII mice that did not receive transplants, lysosomal storage material (arrows) filled the sinus lining cells (A) and osteoblasts (C). The spleen (E) and liver (G) of untreated NOD/SCID/MPSVII mice also contained large amounts of storage material (arrows). In NOD/SCID/MPSVII mice that received human CD34⁺cells, the bone marrow sinus lining cells (B) and osteoblasts (D) were essentially free of lysosomal distention (arrows). The spleen (F) of such treated mice typically showed marked correction with only residual levels of storage material present (arrow). The liver (H) of transplanted NOD/SCID/MPSVII mice showed essentially complete removal of lysosomal storage material. Original magnifications: × 833 (A-D); × 416 (E-H).