144 results on '"Tisdale, John"'
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2. Gene therapy for sickle cell disease
3. Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease
4. Increased Potency and Uniformity of Fetal Hemoglobin Induction from Base Editing Compared to Cas9 Nuclease
5. Phenotypic Screening of the Reframe Drug Repurposing Library to Discover New Drugs for Treating Sickle Cell Disease
6. A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of Mgta-145 in Combination with Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients with Sickle Cell Anemia
7. Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study
8. A macaque clonal hematopoiesis model demonstrates expansion of TET2-disrupted clones and utility for testing interventions
9. Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
10. CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model
11. Optimization of Autologous Hematopoietic Progenitor Stem Cell Apheresis Collection from Plerixafor-Mobilized Patients with Sickle Cell Disease
12. Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)
13. Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models
14. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation
15. Gene therapy for sickle cell disease: moving from the bench to the bedside
16. Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease
17. Dietary iron restriction improves markers of disease severity in murine sickle cell anemia
18. Intrabone transplantation of CD34+ cells with optimized delivery does not enhance engraftment in a rhesus macaque model
19. NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease
20. Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells
21. Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling
22. Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
23. Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
24. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease
25. Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
26. 2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations
27. CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies
28. Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model
29. Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor
30. Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients
31. Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study
32. Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy
33. Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference
34. A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates
35. Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing
36. The Relationships between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy
37. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
38. Reversal of a rheologic cardiomyopathy following hematopoietic stem cell transplantation for sickle cell disease
39. Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34+ Cells from Patients with Sickle Cell Disease
40. Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial
41. Intrabone Delivery of CD34+ Cells Using an Optimized Delivery System Does Not Enhance Engraftment in a Rhesus Macaque Model of Hematopoietic Stem Cell Transplantation
42. Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
43. Pain and Opioid Use after Reversal of Sickle Cell Disease Following Hematopoietic Stem Cell Transplant
44. Bone Marrow Characterization in Sickle Cell Disease: Inflammation and Stress Erythropoiesis Lead to Suboptimal CD34 Recovery Compared to Normal Volunteer Bone Marrow
45. Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease
46. At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT
47. Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT
48. Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease
49. Prevalence of Anti-Human Factor VIII Binding IgG Antibodies in Sickle Cell Disease Patients before and after Hematopoietic Stem Cell Transplantation
50. At Least 20% Donor Myeloid Chimerism Is Necessary to Reverse the Sickle Phenotype after Allogeneic Hematopoietic Stem Cell Transplantation
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