Throughout the 19 days of assessment, Ighmbp2 nmd/nmd + scAAV9- Abt1 (4 × 10 11 ) animals trended to have better HLS then Ighmbp2 nmd/nmd animals, but because of the variation within each group, the deviation did not reach statistical significance.
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ABT1 modifies SMARD1 pathology via interactions with IGHMBP2 and stimulation of ATPase and helicase activity.
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