4 results on '"Zumberge, Nicholas A"'
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2. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect
3. Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB
4. Prospective natural history study of mucopolysaccharidosis types IIIA and IIIB (Sanfilippo syndrome)
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