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- 2015
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Mark
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
(
- Contribution to journal › Article
- 2011
-
Mark
Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia.
(
- Contribution to journal › Article
- 2010
-
Mark
Canonical BMP signaling is dispensable for hematopoietic stem cell function in both adult and fetal liver hematopoiesis, but essential to preserve colon architecture.
(
- Contribution to journal › Article
- 2006
-
Mark
Effective cell and gene therapy in a murine model of Gaucher disease.
(
- Contribution to journal › Article