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- 2023
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Mark
Engineered human Diamond-Blackfan anemia disease model confirms therapeutic effects of clinically applicable lentiviral vector at single-cell resolution
(
- Contribution to journal › Article
- 2022
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Mark
Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector
(
- Contribution to journal › Article
- 2021
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Mark
Gene therapy for infantile malignant osteopetrosis : review of pre-clinical research and proof-of-concept for phenotypic reversal
(
- Contribution to journal › Scientific review
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Mark
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
(
- Contribution to journal › Article
- 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
- 2017
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Mark
Forced expression of human macrophage colony-stimulating factor in CD34+ cells promotes monocyte differentiation in vitro and in vivo but blunts osteoclastogenesis in vitro
2017) In European Journal of Haematology(
- Contribution to journal › Article
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Mark
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
(
- Contribution to journal › Article
- 2015
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Mark
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
(
- Contribution to journal › Article
- 2014
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Mark
Gene therapy cures the anemia and lethal bone marrow failure in mouse model for RPS19-deficient Diamond-Blackfan anemia.
(
- Contribution to journal › Article
- 2013
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Mark
Lentivirus-Induced Dendritic Cells for Immunization Against High-Risk WT1(+) Acute Myeloid Leukemia
(
- Contribution to journal › Article
- 2011
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Mark
Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia.
(
- Contribution to journal › Article
- 2009
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Mark
A Novel Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia Locates the Erythroid Defect at CFU-E/Proerythroblast Transition
(
- Contribution to journal › Published meeting abstract