Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Tardieu, Marc, Zérah, Michel, Husson, Béatrice, de Bournonville, Stéphanie, Deiva, Kumaran, Adamsbaum, Catherine, Vincent, Fanny, Hocquemiller, Michael +9 more·2014·Human Gene Therapy

Cited by (261)

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  8. In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
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  9. AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
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  10. Gene therapy for lysosomal storage diseases and peroxisomal diseases
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  12. Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep
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  13. Gene Therapy for Pompe Disease: The Time is now
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  14. Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice
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  15. A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
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  16. An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA
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  17. Innovative Treatments for Lysosomal Diseases
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  20. Therapeutic Benefit and Gene Network Regulation by Combined Gene Transfer of Apelin, FGF2, and SERCA2a into Ischemic Heart
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  23. Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery
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  46. Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?
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