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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
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Mark
Transcriptome sequencing identifies a noncoding, deep intronic variant in CLCN7 causing autosomal recessive osteopetrosis
(
- 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
-
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
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