Table 1.
Pathological findings in the main non-target tissues.
| Spleen | Lung | Kidney | Heart | |
|---|---|---|---|---|
| AAV2-LDLR | hemosiderin ++ | nd | calcification in tubuli +++ | n/a |
| AAV2-control | hemosiderin + | nd | calcification in tubuli +/++ | nd |
| AAV9-LDLR | hemosiderin +/++ | nd | calcification in tubuli ++/+++ | nd |
| AAV9-control | nd | lymphocytes + | nd | nd |
| LV-LDLR | nd | nd | calcification in tubuli +/++; macrophages, cholesterol crystals, connective tissue (fibrosis) |
nd |
| LV-control | nd | nd | calcification in tubuli +/++; macrophages, cholesterol crystals, connective tissue (fibrosis) |
nd |
Histology of non-target tissues was evaluated from HE-stained sections after the follow-up. Slides were analyzed in a blinded fashion. Changes were scored as nd (not detected), + (mild), ++ (moderate) and +++ (severe) and n/a (not analyzed).