AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn2B/- spinal muscular atrophy mice.

Hum Mol Genet · 2023 · PMC10549791 · PMID 37498175

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highly significantno p-value reported

Bioinformatics analysis revealed a highly significant enrichment of terms relating to ‘abnormal morphology’ and ‘dilated cardiomyopathy’ in reference to ‘diseases & disorders, physiological system development & function’ in both dysregulated datasets.

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