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Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B

Heather Prill,Amanda Luu,Bryan Yip,John Holtzinger,Melanie J. Lo,Terri M. Christianson,Gouri Yogalingam,Mika Aoyagi-Scharber,Jonathan H. LeBowitz,Brett E. Crawford,Roger Lawrence

Molecular Therapy — Methods & Clinical Development（2019）

Cited 12|Views31
Key words
Sanfilippo B,MPS IIIB,BMN 250,NAGLU-IGF2,NAGLU,heparan sulfate,non-reducing end
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