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Potential clinical use of Azacitidine and MEK inhibitor combination therapy in PTPN11-mutated Juvenile myelomonocytic leukemia.
Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction.
Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation.
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.
Gene therapy and bone marrow transplantation for thalassemia: changing of the guard?
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.
Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.
Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy.