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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
- 2013
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Mark
Lentiviral gene transfer of TCIRG1 into peripheral blood CD34(+) cells restores osteoclast function in infantile malignant osteopetrosis.
(
- Contribution to journal › Article
- 2009
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Mark
Neonatal bone marrow transplantation without prior conditioning rapidly reverses osteopetrosis in oc/oc mice despite only minimal donor cell engraftment
(
- Contribution to journal › Published meeting abstract