Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophy
Author:
Affiliation:
1. Center for Gene Therapy, Abigail Wexner Research Institute Nationwide Children's Hospital Columbus Ohio USA
2. Departments of Pediatrics and Neurology Ohio State University Wexner Medical Center Columbus Ohio USA
Publisher
Wiley
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/ppul.25055
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1. Gene Therapy
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5. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1
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