A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. [electronic resource]
Producer: 20161005Description: 228-42 p. digitalISSN:- 2324-8645
- Acetylglucosaminidase -- genetics
- Animals
- Brain -- metabolism
- Dependovirus -- genetics
- Genetic Therapy -- adverse effects
- Genetic Vectors -- administration & dosage
- Liver -- metabolism
- Male
- Mice
- Mice, Inbred C57BL
- Mucopolysaccharidosis III -- therapy
- Organ Specificity
- Practice Guidelines as Topic
- Spinal Cord -- metabolism
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Publication Type: Journal Article
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