Figure 1.

AAV‐MPRO gave only moderate effects on survival, body weight, and motor functions in the severe SMA mice. Severe SMA mice received either saline or a single dose of AAV‐MPRO (2.5 × 10¹⁰ vp) by subcutaneous injection at PND 0. (A) Kaplan–Meier survival curves of saline‐treated (n = 11) and AAV‐MPRO‐treated (n = 9) SMA mice (log‐rank P = 0.0014). (B) Body weight (g) of saline‐treated and AAV‐MPRO‐treated severe SMA mice (n = 4 per group, ^*** P < 0.001, Student's t‐test). (C) The ability of righting reflex in SMA mice received saline or AAV‐MPRO treatment (n = 11 per group). (D) The recorded hind‐limb suspension score in saline and AAV‐MPRO‐treated SMA mice (n = 7 per group).