Figure 5.

Muscle and Peripheral Organs Targeting Following Intra-CSF AAV9 and AAVrh10

(A) GFP expression profiles in NHP diaphragm, gastrocnemius, triceps brachii muscles, heart, and liver following AAVrh10-CBA-GFP LIT-KT delivery and AAV9-CBA-GFP administration via LIT-KT and ICV routes. (a–l) GFP fluorescence, green; wheat germ agglutinin (WGA)-Alexa 555 fluorescent labeling, red. Scale bars, 50 μm. (Am–o) GFP fluorescence, green; unmixed autofluorescence, red. Scale bar, 25 μm. (B) % GFP-positive muscle fibers quantified from diaphragm, gastrocnemius, and triceps brachii muscle sections of NHPs injected with AAVrh10-CBA-GFP LIT-KT (n = 2) and of NHPs injected with AAV9-CBA-GFP administration via the LIT-KT route (n = 4) and ICV delivery (n = 2). Data represented on the graph corresponded to at least 1,000 muscle fibers. (C) % GFP-positive area quantified from heart and liver sections of NHPs injected with AAVrh10-CBA-GFP LIT-KT delivery (n = 2) and of NHPs injected with AAV9-CBA-GFP administration via LIT-KT (n = 4) and ICV routes (n = 2). Data represented on the graph corresponded at least 2 mm² of tissue area investigated, with approximately 1,000 cells explored.