Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. [electronic resource]
Producer: 20140307Description: 913-21 p. digitalISSN:- 1476-5462
- Acetylglucosaminidase -- genetics
- Animals
- Animals, Newborn
- Brain -- metabolism
- Circadian Rhythm
- Dependovirus -- genetics
- Genetic Therapy
- Genetic Vectors
- Humans
- Lentivirus -- genetics
- Liver -- enzymology
- Lung -- enzymology
- Male
- Mice
- Mice, Inbred C57BL
- Mice, Knockout
- Motor Activity
- Mucopolysaccharidosis III -- metabolism
- Myocardium -- enzymology
- Treatment Outcome
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Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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