608 results on '"Nienhuis, Arthur"'
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2. Gene Therapy for the Hemoglobin Disorders: Past, Present, and Future
3. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
4. Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities
5. Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
6. A zinc-finger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts
7. Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector–transduced CD34+ cells
8. Relative Stability of α - and β -Globin Messenger RNAs in Homozygous β + Thalassemia
9. Hemoglobin Switching in Sheep and Goats: Induction of Hemoglobin C Synthesis in Cultures of Sheep Fetal Erythroid Cells
10. Chromosomal Localization of Human β Globin Gene on Human Chromosome 11 in Somatic Cell Hybrids
11. Hemoglobin Switching in Sheep: Only the γ Gene is in the Active Conformation in Fetal Liver but all the β and γ Genes are in the Active Conformation in Bone Marrow
12. Production of Glycosylated Physiologically ``Normal'' Human α 1-antitrypsin by Mouse Fibroblasts Modified by Insertion of a Human α 1-antitrypsin cDNA Using a Retroviral Vector
13. Hemoglobin Synthesis in Somatic Cell Hybrids: Globin Gene Expression in Hybrids between Mouse Erythroleukemia and Human Marrow Cells or Fibroblasts
14. Transfer of Genes into Hematopoietic Cells Using Recombinant DNA Viruses
15. Hemoglobin Synthesis in Somatic Cell Hybrids: Coexpression of Mouse with Human or Chinese Hamster Globin Genes in Interspecific Somatic Cell Hybrids of Mouse Erythroleukemia Cells
16. Recombinant Adeno-Associated Virus (rAAV)-Mediated Expression of a Human γ-Globin Gene in Human Progenitor-Derived Erhythroid Cells
17. Development of a High-Titer Retrovirus Producer Cell Line Capable of Gene Transfer Into Rhesus Monkey Hematopoietic Stem Cells
18. Expression of the Human β -globin Gene Following Retroviral-Mediated Transfer into Multipotential Hematopoietic Progenitors of Mice
19. Combination of Interleukins 3 and 6 Preserves Stem Cell Function in Culture and Enhances Retrovirus-Mediated Gene Transfer into Hematopoietic Stem Cells
20. Mechanism of Kinase Activation in the Receptor for Colony-Stimulating Factor 1
21. Retroviral-Mediated Transfer of Genomic Globin Genes Leads to Regulated Production of RNA and Protein
22. The Interleukin 3 Gene is Located on Human Chromosome 5 and is Deleted in Myeloid Leukemias with a Deletion of 5q
23. Mechanism of DNase I Hypersensitive Site Formation Within the Human Globin Locus Control Region
24. Regulated High Level Expression of a Human γ-Globin Gene Introduced Into Erythroid Cells by an Adeno-Associated Virus Vector
25. A β -globin Gene, Inactive in the K562 Leukemic Cell, Functions Normally in a Heterologous Expression System
26. Chromosomal Organization of the Human Dihydrofolate Reductase Genes: Dispersion, Selective Amplification, and a Novel Form of Polymorphism
27. ``Silent'' Nucleotide Substitution in a β + -thalassemia Globin Gene Activates Splice Site in Coding Sequence RNA
28. Acquired α -thalassemia in Preleukemia is due to Decreased Expression of All Four α -globin Genes
29. RNA Processing Errors in Patients with β -thalassemia
30. Intronless Human Dihydrofolate Reductase Genes are Derived from Processed RNA Molecules
31. Hemoglobin Switching in Sheep and Goats. VI. Commitment of Erythroid Colony-Forming Cells to the Synthesis of BetaC Globin
32. Hemoglobin Switching in Sheep and Goats. V. Effect of Erythropoietin Concentration on in Vitro Erythroid Colony Growth and Globin Synthesis
33. Selection of Drug-Resistant Bone Marrow Cells in Vivo After Retroviral Transfer of Human MDR1
34. COOH-Terminal--Modified Interleukin-3 Is Retained Intracellularly and Stimulates Autocrine Growth
35. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
36. Hemoglobin Switching in Sheep and Goats: Change in Functional Globin Messenger RNA in Reticulocytes and Bone Marrow Cells
37. Hemogloblin Switching in Sheep and Goats: Erythropoietin-Dependent Synthesis of Hemoglobin C in Goat Bone-Marrow Cultures
38. An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation
39. A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells
40. C1-6 AAV-mediated gene therapy for haemophilia B
41. Autocrine expression of both endostatin and green fluorescent protein provides a synergistic antitumor effect in a murine neuroblastoma model
42. Regulation of Globin Gene Expression
43. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
44. High level of fetal-globin reactivation by designed transcriptional activator-like effector
45. Ninth Cooleyʼs Anemia Symposium: summary and perspective
46. SUPPRESSION OF CLONAL DOMINANCE IN CULTURED HUMAN LYMPHOID CELLS BY ADDITION OF THE CHS4 INSULATOR TO A LENTIVIRAL VECTOR: O10
47. Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia
48. Eighth Cooley's Anemia Symposium: Summation and Perspective
49. Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus–based lentiviral vector system
50. Hemoglobin Switching in Sheep and Goats: Occurrence of Hemoglobins A and C in the Same Red Cell
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