scholarly journals 873. Synergistic and Antagonistic Effects of Neonatal Intracranial AAV Gene Therapy and Bone Marrow Transplantation in Sanfilippo Syndrome Type B Mice

2008 ◽  
Vol 16 ◽  
pp. S325-S326
Keyword(s):  
Gene Therapy ◽  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome ◽  
Antagonistic Effects ◽  
B Mice

scholarly journals Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice

Gene Therapy ◽  
2013 ◽  
Vol 20 (9) ◽  
pp. 913-921 ◽  
Author(s):  
C D Heldermon ◽  
E Y Qin ◽  
K K Ohlemiller ◽  
E D Herzog ◽  
J R Brown ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome ◽  
B Mice ◽  
Lentiviral Gene Therapy

Neural stem cells provide continuous enzyme replacement therapy and reduce neuropathology in Sanfilippo syndrome type B mice

2018 ◽  
Vol 123 (2) ◽  
pp. S115
Author(s):  
Yewande E.O. Pearse ◽  
Don Clarke ◽  
Kan Shih-hsin ◽  
Steven Q. Le ◽  
Valentina Sanghez ◽  
...  
Keyword(s):  
Stem Cells ◽  
Enzyme Replacement Therapy ◽  
Neural Stem Cells ◽  
Replacement Therapy ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome ◽  
Enzyme Replacement ◽  
B Mice
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