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- 2018
-
Mark
Fetal gene therapy for neurodegenerative disease of infants
(
- Contribution to journal › Letter
-
Mark
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
(
- Contribution to journal › Article
- 2013
-
Mark
Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes
(
- Contribution to journal › Article
- 2009
-
Mark
Fetal Gene Therapy for Acute Neuronopathic Gaucher Disease
2009) 12th Annual Meeting of the American Society of Gene Therapy In Molecular Therapy 17. p.357-357(
- Contribution to journal › Published meeting abstract