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- 2017
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Mark
Roscoe Owen Brady, MD : Remembrances of co-investigators and colleagues
(
- Contribution to journal › Article
- 2015
-
Mark
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
(
- Contribution to journal › Article
- 2010
-
Mark
Development of Novel Therapies in Murine Models for Gaucher Disease
2010) 7th Annual Conference of the British-Society-for-Gene-Therapy In Human Gene Therapy 21(4). p.490-491(
- Contribution to journal › Published meeting abstract
-
Mark
Successful Treatment of Metachromatic Leukodystrophy Using Bone Marrow Transplantation of HoxB4 Overexpressing Cells
(
- Contribution to journal › Article
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Mark
Altered expression and distribution of cathepsins in neuronopathic forms of Gaucher disease and in other sphingolipidoses
(
- Contribution to journal › Article
- 2009
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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
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Mark
No evidence for activation of the unfolded protein response in neuronopathic models of Gaucher disease
(
- Contribution to journal › Article
- 2005
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Mark
Development of cellular models for ribosomal protein S19 (RPS19)-deficient diamond-blackfan anemia using inducible expression of siRNA against RPS19.
(
- Contribution to journal › Article
- 2004
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Mark
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences
(
- Contribution to journal › Scientific review
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Mark
Functionally distinct subpopulations of cord blood CD34+ cells are transduced by adenoviral vectors with serotype 5 or 35 tropism.
(
- Contribution to journal › Article