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16. Hematopoietic Stem Cell Gene Therapy For Wiskott- Aldrich Syndrome

17. Investigating Synthetic Lethality Between mTOR Hyperactivation and Cap-Dependent mRNA Translation In a Receptor Tyrosine Kinase Driven Model Of Acute T-Cell Leukemia

21. EVI-1 Mediates Apoptosis Resistance Via CD261 Induction and Enhances Leukemogenic Potential in Human Acute Lymphoblastic Leukemia

22. Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

26. Generation of a Transgenic Mouse with Inducible Constitutively Active Stat5a,

27. Use of the in Vitro Immortalization Assay to Quantify the Impact of Integration Spectrum and Vector Design on Insertional Mutagenesis

28. Efficacy of Gene Therapy for Wiskott-Aldrich-Syndrome

31. Evidence for Cooperation of Receptor Tyrosine Kinases and Activating NOTCH Mutations to Hyperactivate mTOR in T-Cell Leukemia: A Rationale Basis for Targeted Therapy

40. Neurotrophin Receptors and Ligands Are Highly Expressed in Acute Leukemia and Promote Leukemogenesis.

41. Retroviral vector insertion sites associated with dominant hematopoietic clones mark “stemness” pathways

42. Leukemogenic Cascade Induced by a Constitutively Active Neurotrophin Receptor, ΔTrkA.

46. In Vivo Chemotherapy Protection and Efficacy of Multidrug Resistance (MDR-1) Gene Transfer in a Patient with Refractory Non-Hodgkin’s Lymphoma (NHL).

47. Hematopoietic Repopulation, In Vivo, with Genetically Defined Clones Derived from HOXB4 Expressing ES-Cells.

48. LEF-1 Transcription Factor Regulates Proliferation and Differentiation of Myeloid Progenitors in Healthy Individuals and in Patients with Severe Congenital Neutropenia (CN).

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