Heldermon, C D <br/><br/>
Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.  [electronic resource] 

 -  Gene therapy  Sep 2013 
 - 913-21 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1476-5462 
<br/><br/><label>Standard No.: </label>10.1038/gt.2013.14  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Acetylglucosaminidase--genetics<br/>Animals<br/>Animals, Newborn<br/>Brain--metabolism<br/>Circadian Rhythm<br/>Dependovirus--genetics<br/>Genetic Therapy<br/>Genetic Vectors<br/>Humans<br/>Lentivirus--genetics<br/>Liver--enzymology<br/>Lung--enzymology<br/>Male<br/>Mice<br/>Mice, Inbred C57BL<br/>Mice, Knockout<br/>Motor Activity<br/>Mucopolysaccharidosis III--metabolism<br/>Myocardium--enzymology<br/>Treatment Outcome