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1. Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB.

Author

McBride, Kim L., Truxal, Kristen, McNally, Kelly, Kunkler, Krista, Sherrod-Canaan, Tamar, Aylward, Shawn, Miller, Tim, Zumberge, Nicholas, Martin, Lisa, Corridore, Marco, McKee, Christopher, McCarty, Douglas, Fu, Haiyan, and Flanigan, Kevin

Subjects
*GENETIC transformation, *CLINICAL trials, *SANFILIPPO syndrome, *MUCOPOLYSACCHARIDOSIS, *CHILDREN'S health, *DIAGNOSIS, *THERAPEUTICS
Published
2016
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