Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

Clinical trials have demonstrated the potential of ex vivo hematopoietic stem cell gene therapy to treat X-linked severe combined immunodeficiency (SCID-X1) using γ-retroviral vectors, leading to immune system functionality in the majority of treated patients without pretransplant conditioning. The...

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Veröffentlicht in:	Molecular therapy 2011-10, Vol.19 (10), p.1867-1877
Hauptverfasser:	Huston, Marshall W, van Til, Niek P, Visser, Trudi P, Arshad, Shazia, Brugman, Martijn H, Cattoglio, Claudia, Nowrouzi, Ali, Li, Yuedan, Schambach, Axel, Schmidt, Manfred, Baum, Christopher, von Kalle, Christof, Mavilio, Fulvio, Zhang, Fang, Blundell, Mike P, Thrasher, Adrian J, Verstegen, Monique MA, Wagemaker, Gerard
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Antibody Formation B-Lymphocytes - immunology Clinical trials Codon Cytokines Gene therapy Genetic Therapy Hematology Hematopoietic Stem Cell Transplantation Immunoglobulins Immunology Infections Interleukin Receptor Common gamma Subunit - genetics Kinases Lentivirus - genetics Lymphocytes Mice Mice, SCID Original Protein expression Proteins Receptors, Antigen, T-Cell - immunology Severe Combined Immunodeficiency - immunology Severe Combined Immunodeficiency - therapy Spleen Stem cells T-Lymphocytes - immunology Transplants & implants Vectors (Biology)
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