Brian Bigger , Stuart M. Ellison , Shaun R. Wood , Rebecca Holley
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Long-term HSC gene therapy in mucopolysaccharidosis type IIIB mice corrects disease with no evidence of insertional mutagenesis despite high vector copy numbers
期刊介绍:
Molecular Genetics and Metabolism contributes to the understanding of the metabolic and molecular basis of disease. This peer reviewed journal publishes articles describing investigations that use the tools of biochemical genetics and molecular genetics for studies of normal and disease states in humans and animal models.