CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis.

However, heterozygous Chp1 mutation had no significant influence on mean muscle fibre size, but showed a clear trend towards a reduced number of small-sized muscle fibres (<300 μm 2 ) and an increased number of medium-sized muscle fibres (300–500 μm 2 ) in SMA- Chp1 vac/ wt + ASO versus SMA + ASO mice at postnatal Day 21 ( Fig. 5 D).

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