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- 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
- 2020
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Mark
Generation of gene-corrected functional osteoclasts from osteopetrotic induced pluripotent stem cells
(
- 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
- 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
- 2010
-
Mark
Genetic analysis of dasatinib-treated chronic myeloid leukemia rapidly developing into acute myeloid leukemia with monosomy 7 in Philadelphia-negative cells.
(
- Contribution to journal › Article
- 2008
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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
- 2007
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Mark
Fusion gene-mediated truncation of RUNX1 as a potential mechanism underlying disease progression in the 8p11 myeloproliferative syndrome.
(
- 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
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Mark
Gene Transfer to Repopulating Human CD34(+) Cells Using Amphotropic-, GALV-, or RD114-Pseudotyped HIV-1-Based Vectors from Stable Producer Cells.
(
- Contribution to journal › Article
- 2003
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Mark
Proliferation deficiency of multipotent hematopoietic progenitors in ribosomal protein S19 (RPS19)-deficient Diamond-Blackfan anemia improves following RPS19 gene transfer
(
- Contribution to journal › Article