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- 2022
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Mark
Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector
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- Contribution to journal › Article
- 2021
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Mark
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
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- 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
- 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
- 2017
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Mark
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
(
- Contribution to journal › Article
- 2015
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Mark
Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
(
- Contribution to journal › Article
- 2014
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Mark
Gene therapy cures the anemia and lethal bone marrow failure in mouse model for RPS19-deficient Diamond-Blackfan anemia.
(
- Contribution to journal › Article