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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
- 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
Regulation and Function of Lentiviral Vector-Mediated TCIRG1 Expression in Osteoclasts from Patients with Infantile Malignant Osteopetrosis : Implications for Gene Therapy
(
- Contribution to journal › Article
-
Mark
Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease : a multicenter experience
(
- Contribution to journal › Article
- 2013
-
Mark
Lentiviral gene transfer of TCIRG1 into peripheral blood CD34(+) cells restores osteoclast function in infantile malignant osteopetrosis.
(
- Contribution to journal › Article