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- 2019
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Mark
Hematopoietic stem cell-targeted neonatal gene therapy with a clinically applicable lentiviral vector corrects osteopetrosis in oc/oc Mice
(
- Contribution to journal › Article
- 2018
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Mark
Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis
(
- Contribution to journal › Article
- 2016
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Mark
Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis : Dose-Dependent Expression and Improved Retinal Structure and Function
(
- Contribution to journal › Article
- 2013
-
Mark
Lentivirus-Induced Dendritic Cells for Immunization Against High-Risk WT1(+) Acute Myeloid Leukemia
(
- Contribution to journal › Article
- 2011
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Mark
Targeting Expression of the Leukemogenic PML-RAR alpha Fusion Protein by Lentiviral Vector-Mediated Small Interfering RNA Results in Leukemic Cell Differentiation and Apoptosis
(
- 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
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Mark
A Human Model of IMO by Lentiviral Mediated shRNA Knock-down of Tcirg1 in CD34+Cells
2010) 18th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy In Human Gene Therapy 21(10). p.1458-1459(
- Contribution to journal › Published meeting abstract
- 2008
-
Mark
Neonatal hematopoietic stem cell transplantation following low-dose busulphan conditioning reverses osteopetrosis and preserves vision in oc/oc mice
2008) 16th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy In Human Gene Therapy 19(10). p.1183-1184(
- Contribution to journal › Published meeting abstract
- 2004
-
Mark
Regulated delivery of glial cell line-derived neurotrophic factor into rat striatum, using a tetracycline-dependent lentiviral vector.
(
- Contribution to journal › Article
- 2002
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Mark
Efficient Oncoretroviral Transduction of Extended Long-Term Culture-Initiating Cells and NOD/SCID Repopulating Cells: Enhanced Reconstitution with Gene-Marked Cells Through an Ex Vivo Expansion Approach.
(
- Contribution to journal › Article