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- 2021
-
Mark
Gene therapy for infantile malignant osteopetrosis : review of pre-clinical research and proof-of-concept for phenotypic reversal
(
- Contribution to journal › Scientific review
- 2016
-
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
- 2007
-
Mark
Murine models of acute neuronopathic Gaucher disease
(
- Contribution to journal › Article
- 2003
-
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
- 2001
-
Mark
Clinical gene therapy in hematology: Past and future
(
- Contribution to journal › Article