37 results on '"Carbonaro, Denise"'
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2. Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency
3. Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction
4. Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
5. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion
6. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates
7. In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency
8. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34+ cells
9. Neonatal Gene Therapy of MPS I Mice by Intravenous Injection of a Lentiviral Vector
10. Efficient Characterization of Retro-, Lenti-, and Foamyvector-Transduced Cell Populations by High-Accuracy Insertion Site Sequencing
11. Gene Therapy for Pediatric AIDS
12. Expression from Second-Generation Feline Immunodeficiency Virus Vectors Is Impaired in Human Hematopoietic Cells
13. A Clinical Trial of Retroviral-Mediated Transfer of arev-Responsive Element Decoy Gene Into CD34+Cells From the Bone Marrow of Human Immunodeficiency Virus-1–Infected Children
14. Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency
15. Towards a phase I clinical trial for autologous hematopoietic stem cells transplantation in cystinosis
16. 32. Towards a Phase I Clinical Trial for Cystinosis
17. 84. Pharmacology/Toxicology Studies for Gene-Modified Hematopoietic Stem Cell Transplantation for Cystinosis
18. 292. Lentivirus Vector Mediated Gene Correction in Artemis-Deficient Severe Combined Immunodeficiency
19. 30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector
20. C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency
21. Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
22. Comparative Results of Gene Therapy for Adenosine Deaminase Deficiency with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy.
23. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report
24. Gene Therapy for Pediatric AIDS
25. 1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in γc Gene Knock-Out Mice
26. 1090. Enzyme Replacement Therapy with Pegylated Adenosine Deaminase (PEG-ADA) Does Not Impede Immune Reconstitution Following Transplantation of Gene-Corrected Bone Marrow Cells in the Murine Model of ADA-SCID
27. High-Resolution Analysis of Cytosine Methylation in the 5′ Long Terminal Repeat of Retroviral Vectors
28. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
29. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates.
30. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.
31. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report
32. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+cells in ADA-deficient SCID neonates
33. The 24-hour time budget of Przewalski horses
34. Altered reactivity of the rat adrenal medulla
35. Night-time behavior of stabled and pastured peri-parturient ponies
36. 37. A Novel Form of Enzyme Replacement Therapy for ADA-Deficiency: In Vivo Transduction by Neonatal Injection of Lentivirus Expressing ADA
37. 805. Effective Suicide Gene Therapy of Leukemia in a Novel Model of Retroviral Insertion
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