Skip to main content
. Author manuscript; available in PMC: 2018 Jan 1.
Published in final edited form as: J Neuromuscul Dis. 2017;4(1):59–66. doi: 10.3233/JND-170209

Figure 1.

Figure 1

Illustration of experimental groups and longitudinal timelines in the study. The scAAV9-shRNA against pSMN or scrambled-shRNA was injected into five-day old piglets to generate a large-animal SMA model. The early-treatment group (shRNA/Early) consists of knockdown piglets receiving a second intrathecal injection 24 hours later with the rescue vector scAAV9-SMN. In the late-treatment group (shRNA/Late), SMA animals received the second intrathecal injection of scAAV9-SMN at the onset of symptoms at around postnatal day (PND) 33–36. Blood was withdrawn pre-weaning, post-weaning and every week thereafter till the end-point of the study.