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Bringing gene therapy based SOD1 silencing towards human trials: A highly efficacious, off-target free and biomarker supported strategy for fALS
Published on Jul 21, 2017757 Views
Aims: 1. To evaluate the therapeutic efficacy of scAAV9-shRNA mediated SOD1 silencing in the SOD1-G93A mouse model using a clinic ready vector. 2. To investigate the measurement of CSF SOD1 protein
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Chapter list
Bringing Gene therapy based SOD1 silencing towards clinical trials00:00
Study aims00:13
Background00:45
Clinic-ready vector design01:18
In Vivo Efficacy01:47
Route of injection01:59
P1 – neuroscoring and onset - 102:28
P1 – neuroscoring and onset - 202:40
P1 – neuroscoring and onset - 302:56
P1 – rotarod and survival03:11
P1 mice – 144 days old03:54
Treatment rescues motor neurons04:20
Treatment reduces reactive gliosis04:34
P40 – neuroscoring and rotarod04:49
P40 – survival05:07
Measurement of SOD1 depletion in the CSF05:16
Cerebrospinal Fluid05:40
In vitro investigation of off-target effects06:01
Seed regions in 3’UTRs06:24
RISC06:53
Construct validation07:59
Genes differentially expressed from Negative control08:36
Summary09:17
Acknowledgements09:41
Thank you for your attention !09:57