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2. Severe Combined Immunodeficiencies in Humans

3. LentiGlobin gene therapy for transfusion-dependent β-Thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study

4. Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

5. Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

7. Intensive and Prolonged Treatment of Focal and Segmental Glomerulosclerosis Recurrence in Adult Kidney Transplant Recipients: A Pilot Study

10. 58: Risk Factor Analysis of Outcomes After Unrelated Cord Blood Transplantation for Children with Hurlers Syndrome. An EUROCORD-Duke University Collaborative Study

11. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure

14. The VAD chemotherapy regimen plus a G-CSF dose of 10 μg/kg is as effective and less toxic than high-dose cyclophosphamide plus a G-CSF dose of 5 μg/kg for progenitor cell mobilization: results from a monocentric study of 82 patients

16. Gene Therapy for Severe Combined Immunodeficiency X1.

17. Adoptive Immunotherapy with Donor Allodepleted T Cells.

21. Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors

22. LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

44. In VivoInfusion of Anti-LFA-1 and Anti-CD2 Antibodies Prevents Graft Failure After HLA Partially Incompatible Bone Marrow Transplantation in Children with High Risk Acute Lymphoblastic Leukaemia

48. A phase II trial of partially incompatible bone marrow transplantation for high-risk acute lymphoblastic leukaemia in children: prevention of graft rejection with anti-LFA-1 and anti-CD2 antibodies

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