798 results on '"Bueren, Juan A."'
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2. Chapter From the Molecular Biology to the Gene Therapy of a DNA Repair Syndrome: Fanconi Anemia
3. Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I
4. Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells
5. Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia
6. Access to gene therapy for rare diseases when commercialization is not fit for purpose
7. Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment
8. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes
9. Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
10. Mesenchymal stem/stromal cell-based therapy for the treatment of rheumatoid arthritis: An update on preclinical studies
11. In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9
12. Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
13. Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization
14. Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1
15. Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10
16. NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia
17. Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
18. Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors
19. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
20. Non-Homologous End-Joining Defect in Fanconi Anemia Hematopoietic Cells Exposed to Ionizing Radiation
21. A New Approach to Evaluate the Total Reserve of Hematopoietic Progenitors after Acute Irradiation
22. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases
23. Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent
24. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients
25. The downregulated membrane expression of CD18 in CD34+ cells defines a primitive population of human hematopoietic stem cells
26. TALEN mediated gene editing in a mouse model of Fanconi anemia
27. Detectable clonal mosaicism in blood as a biomarker of cancer risk in Fanconi anemia
28. Severe Leukocyte Adhesion Deficiency-I (LAD-I) Lentiviral-Mediated Ex-Vivo Gene Therapy: Ongoing Phase 1/2 Study Results
29. Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency
30. Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV)
31. Supplementary Materials for Gene Therapy Restores the Transcriptional Program of Hematopoietic Stem Cells in Fanconi Anemia [Dataset]
32. Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides
33. Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia
34. Supplementary Fig. S3 from Relevance of the Fanconi anemia pathway in the response of human cells to trabectedin
35. Data from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
36. Supplementary Figure 7 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
37. Supplementary Figure 3 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
38. Supplementary Figure 1 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
39. Data from Relevance of the Fanconi anemia pathway in the response of human cells to trabectedin
40. Supplementary Figure 5 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
41. Supplementary Figure 8 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
42. Supplementary Figure 2 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
43. Supplementary Text from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
44. Supplementary Figure 4 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
45. Supplementary Figure 6 from Gefitinib and Afatinib Show Potential Efficacy for Fanconi Anemia–Related Head and Neck Cancer
46. Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model
47. Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
48. Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials
49. Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study
50. Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells
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