Table 6.
Summary of Tissue Pathology Findings 3 Months after Treatment with rAAVrh74.MCK.GALGT2
| Subject (Total Dose) | 07D185 (Control) | 07D147 (5E+13 vg/kg) | 04C034 (6E+13 vg/kg) |
|---|---|---|---|
| Femoral artery left and right | – | – | – |
| Sural artery left and right | – | – | – |
| Gonad | – (testis) | – (testis) | – (ovary) |
| Kidney | infiltration, lymphocytic, perivascular, focal, mild, cortex | – | – |
| Urinary bladder | – | infiltrate, mononuclear cell, focal, minimal | – |
| Lung | – | – | infiltrate, mononuclear cell, multifocal, minimal |
| Pancreas | – | – | – |
| Spleen | – | – | – |
| Stomach | – | – | inflammation, plasmacytic, diffuse, mild |
| Liver | – | infiltrate, mononuclear cell, multifocal, minimal | vacuolation, hepatocellular, diffuse, moderate (consistent with glycogen) |
| Hearta | – | – | left ventricle, infiltrate, mononuclear cell, multifocal, minimal, |
| Diaphragm | – | – | – |
| Skeletal muscleb | – | – | – |
| Skeletal musclec | – | – | infiltrate, mixed cell (macrophages mainly), multifocal, minimal |
| Skeletal muscled | – | – | infiltrate, mononuclear cell, focal, minimal |
Lesion scores: dash, within normal limits.
a
Atria and ventricles (free walls).
b
Biceps brachii, biceps femoris, extensor digitorum longus, gastrocnemius (medial and lateral), gluteus (maximus, medius, and minimus), gracilis, rectus femoris, sartorius, semimembranosus, semitendinosus, soleus, triceps brachii, tibialis anterior, and vastus (intermedius, lateralis, and medialis).
c
Right biceps femoris.
d
Right gracilis.