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

Autor: Brown, Sharon J, Šoltić, Darija, Synowsky, Silvia A, Shirran, Sally L, Chilcott, Ellie, Shorrock, Hannah K, Gillingwater, Thomas H, Yáñez-Muñoz, Rafael J, Schneider, Bernard, Bowerman, Melissa, Fuller, Heidi R
Zdroj: Human Molecular Genetics; 10/15/2023, Vol. 32 Issue 20, p2950-2965, 16p
Databáze: Complementary Index