AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
Aurore Besse, Stéphanie Astord, Thibaut Marais, Marianne Roda, Benoit Giroux, François‐Xavier Lejeune, Frédéric Relaix, Piera Smeriglio, Martine Barkats, Maria Grazia Biferi
Topics & Concepts
Spinal muscular atrophyPhenotypeAtrophyBiologyNeurosciencePathologyCell biologyAnatomyMedicineGeneGeneticsNeurogenetic and Muscular Disorders ResearchMuscle Physiology and DisordersCongenital Anomalies and Fetal Surgery