Meadows, Aaron S <br/><br/>
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.  [electronic resource] 

 -  Human gene therapy. Clinical development  Dec 2015 
 - 228-42 p.  digital 
<br/><br/> Publication Type: Journal Article 
<br/><br/><label>ISSN: </label>2324-8645 
<br/><br/><label>Standard No.: </label>10.1089/humc.2015.132  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Acetylglucosaminidase--genetics<br/>Animals<br/>Brain--metabolism<br/>Dependovirus--genetics<br/>Genetic Therapy--adverse effects<br/>Genetic Vectors--administration & dosage<br/>Liver--metabolism<br/>Male<br/>Mice<br/>Mice, Inbred C57BL<br/>Mucopolysaccharidosis III--therapy<br/>Organ Specificity<br/>Practice Guidelines as Topic<br/>Spinal Cord--metabolism