Fig. 4 |. Focal expression of μDystro, but not μutro, elicits a detectable peripheral and local immune response in the GSHP dystrophin deletional-null dog model.

a, Experimental timeline. Each dog (Grinch and Ned) received intra-muscular (IM) injections of both AAV-μDystro (right) and AAV-μUtro (left) at equivalent doses (1 × 10¹² vg kg⁻¹) into their tibialis anterior compartment. b, Peripheral blood mononuclear cells (PBMCs) collected pre- and two, four, six and eight weeks post-injection were cultured with synthetic peptides spanning the entire μDystro (pool A–D) and μUtro (pool E–J) peptide sequences, while vaccine peptides and phorbol myristate acetate (PMA)/ionomyocin served as positive controls. A positive result is interpreted as ≥5 spot forming units (SFU) per 1 × 10⁵ PBMCs (dotted line). c, Immunofluorescence staining of muscle biopsies collected four weeks post-injection against utrophin (green) and dystrophin (red). Experiments were repeated independently at least two times with similar results. d, Representative H&E stain of muscle biopsies collected four weeks post-injection (see also Extended Data Fig. 9a). Experiments were repeated independently at least two times with similar results. (See Source Data Fig. 4).