Concepedia

SGT-001 Micro-dystrophin gene therapy for Duchenne muscular dystrophy

Joel Schneider, Jorge González, Kristy J. Brown, Diane Golebiowski, Valeria Ricotti, Jorge A. Quiroz, Carl Morris

Neuromuscular Disorders · 2017 · 20 citations · 0 references

Concepts

MedicineGeneticsMolecular GeneticsGene DeliveryGene TherapyMedical GeneticsGenetic DisordersIn Vivo Gene TherapyMuscle DisordersMuscle PathologyMuscular DystrophyNeuromuscular JunctionDystrophin DeficiencyEditing For Muscular DystrophyMuscle Gene TherapyMuscular Dystrophy GeneticsMuscular Dystrophy Pathophysiology