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Gene therapy of X-linked severe combined immunodeficiency.
- Published in:
- 2002
- By:
- Publication type:
- journal article
Gene therapy for severe combined immunodeficiency: are we there yet?
- Published in:
- 2007
- By:
- Publication type:
- journal article
A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome.
- Published in:
- 2007
- By:
- Publication type:
- journal article
Severe combined immunodeficiency caused by deficiency in either the δ or the ε subunit of CD3.
- Published in:
- Journal of Clinical Investigation, 2004, v. 114, n. 10, p. 1512, doi. 10.1172/JCI200422588
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- Publication type:
- Article
A method to sequence and quantify DNA integration for monitoring outcome in gene therapy.
- Published in:
- Nucleic Acids Research, 2011, v. 39, n. 11, p. e72, doi. 10.1093/nar/gkr140
- By:
- Publication type:
- Article
DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer.
- Published in:
- Nucleic Acids Research, 2008, v. 36, n. 9, p. e49, doi. 10.1093/nar/gkn125
- By:
- Publication type:
- Article
Amniotic Fluid Stem Cells Restore the Muscle Cell Niche in a HSA-Cre, Smn<sup>F7/F7</sup> Mouse Model.
- Published in:
- Stem Cells, 2012, v. 30, n. 8, p. 1675, doi. 10.1002/stem.1134
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- Publication type:
- Article
Human T-Lymphoid Progenitors Generated in a Feeder-Cell-Free Delta-Like-4 Culture System Promote T-Cell Reconstitution in NOD/SCID/γc<sup>−/−</sup> Mice.
- Published in:
- Stem Cells, 2012, v. 30, n. 8, p. 1771, doi. 10.1002/stem.1145
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- Publication type:
- Article
Bone Marrow Transplantation Attenuates the Myopathic Phenotype of a Muscular Mouse Model of Spinal Muscular Atrophy.
- Published in:
- Stem Cells, 2006, v. 24, n. 12, p. 2723, doi. 10.1634/stemcells.2006-0170
- By:
- Publication type:
- Article
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.
- Published in:
- Nature, 2010, v. 467, n. 7313, p. 318, doi. 10.1038/nature09328
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- Publication type:
- Article
Gene therapy: X-SCID transgene leukaemogenicity.
- Published in:
- Nature, 2006, v. 443, n. 7109, p. E5, doi. 10.1038/nature05219
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- Publication type:
- Article
Opinion: Gene therapy of severe combined immunodeficiencies.
- Published in:
- Nature Reviews Immunology, 2002, v. 2, n. 8, p. 615, doi. 10.1038/nri859
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- Publication type:
- Article
Severe combined immunodeficiency. A model disease for molecular immunology and therapy.
- Published in:
- Immunological Reviews, 2005, v. 203, n. 1, p. 98, doi. 10.1111/j.0105-2896.2005.00223.x
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- Publication type:
- Article
Gene therapy of severe combined immunodeficiencies.
- Published in:
- Immunological Reviews, 2000, v. 178, n. 1, p. 13, doi. 10.1034/j.1600-065x.2000.17806.x
- By:
- Publication type:
- Article
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.
- Published in:
- 2005
- By:
- Publication type:
- journal article
Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia.
- Published in:
- Medical Journal of Australia, 2005, v. 182, n. 9, p. 458, doi. 10.5694/j.1326-5377.2005.tb06785.x
- By:
- Publication type:
- Article
20 years of gene therapy for SCID.
- Published in:
- 2010
- By:
- Publication type:
- Opinion
Shortening the immunodeficient period after hematopoietic stem cell transplantation.
- Published in:
- Immunologic Research, 2009, v. 44, n. 1-3, p. 54, doi. 10.1007/s12026-008-8080-7
- By:
- Publication type:
- Article
The future of gene therapy.
- Published in:
- 2004
- By:
- Publication type:
- Book Review
A specific time course for mobilization of peripheral blood CD34+ cells after plerixafor injection in very poor mobilizer patients: impact on the timing of the apheresis procedure.
- Published in:
- Transfusion, 2013, v. 53, n. 3, p. 564, doi. 10.1111/j.1537-2995.2012.03744.x
- By:
- Publication type:
- Article
Evaluation of an algorithm based on peripheral blood hematopoietic progenitor cell and CD34+ cell concentrations to optimize peripheral blood progenitor cell collection by apheresis.
- Published in:
- Transfusion, 2007, v. 47, n. 10, p. 1851, doi. 10.1111/j.1537-2995.2007.01407.x
- By:
- Publication type:
- Article
Gene therapy of severe combined immunodeficiencies.
- Published in:
- Journal of Gene Medicine, 2001, v. 3, n. 3, p. 201, doi. 10.1002/1521-2254(200105/06)3:3<201::AID-JGM195>3.0.CO;2-Z
- By:
- Publication type:
- Article
Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 11, p. 2031, doi. 10.1038/mt.2011.178
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- Publication type:
- Article
Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 7, p. 1193, doi. 10.1038/mt.2011.78
- By:
- Publication type:
- Article
Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 5, p. 965, doi. 10.1038/mt.2010.50
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- Publication type:
- Article
Stable and Functional Lymphoid Reconstitution in Artemis-deficient Mice Following Lentiviral Artemis Gene Transfer Into Hematopoietic Stem Cells.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 8, p. 1490, doi. 10.1038/mt.2008.118
- By:
- Publication type:
- Article
Restoration of Human B-cell Differentiation Into NOD-SCID Mice Engrafted With Gene-corrected CD34<sup>+</sup> Cells Isolated From Artemis or RAG1-deficient Patients.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 2, p. 396, doi. 10.1038/sj.mt.6300353
- By:
- Publication type:
- Article
660. The Effect of the B2 Mutation on Hematopoietic Reconstitution in the French X-SCID Gene Therapy Trial.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S254, doi. 10.1016/j.ymthe.2006.08.737
- By:
- Publication type:
- Article
Transduced CD34<sup>+</sup> cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice
- Published in:
- Molecular Therapy, 2003, v. 7, n. 3, p. 317, doi. 10.1016/S1525-0016(03)00002-9
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- Publication type:
- Article
Haematopoietic stem cell transplantation for SCID patients: where do we stand?
- Published in:
- British Journal of Haematology, 2013, v. 160, n. 2, p. 146, doi. 10.1111/bjh.12119
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- Publication type:
- Article
CD34<sup>+</sup> stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies.
- Published in:
- British Journal of Haematology, 2006, v. 135, n. 4, p. 533, doi. 10.1111/j.1365-2141.2006.06333.x
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- Publication type:
- Article
Human leucocyte antigen-identical haematopoietic stem cell transplantation in major histocompatiblity complex class II immunodeficiency: reduced survival correlates with an increased incidence of acute graft- versus-host disease and pre-existing viral infections
- Published in:
- British Journal of Haematology, 2006, v. 134, n. 5, p. 510, doi. 10.1111/j.1365-2141.2006.06213.x
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- Publication type:
- Article
IL-7 effect on immunological reconstitution after HSCT depends on MHC incompatibility.
- Published in:
- British Journal of Haematology, 2004, v. 126, n. 6, p. 844, doi. 10.1111/j.1365-2141.2004.05134.x
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- Publication type:
- Article
Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous ATR Mutations.
- Published in:
- Human Mutation, 2013, v. 34, n. 2, p. 374, doi. 10.1002/humu.22245
- By:
- Publication type:
- Article
First experience of autologous peripheral blood stem cell mobilization with biosimilar granulocyte colony-stimulating factor.
- Published in:
- 2011
- By:
- Publication type:
- Journal Article
First experience of autologous peripheral blood stem cell mobilization with biosimilar granulocyte colony- stimulating factor.
- Published in:
- Advances in Therapy, 2011, v. 28, n. 4, p. 304, doi. 10.1007/s12325-011-0009-1
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- Publication type:
- Article
Integration of Retroviruses: A Fine Balance between Effi ciency and Danger.
- Published in:
- PLoS Medicine, 2005, v. 2, n. 1, p. 22, doi. 10.1371/journal.pmed.0020010
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- Publication type:
- Article
Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV.
- Published in:
- European Journal of Immunology, 2006, v. 36, n. 1, p. 224, doi. 10.1002/eji.200535401
- By:
- Publication type:
- Article
Characterization of antigen-specific repertoire diversity following in vitro restimulation by a recombinant adenovirus expressing human cytomegalovirus pp65.
- Published in:
- European Journal of Immunology, 2003, v. 33, n. 3, p. 760, doi. 10.1002/eji.200323628
- By:
- Publication type:
- Article
Correction of Fas (CD95) deficiency by haploidentical bone marrow transplantation.
- Published in:
- European Journal of Immunology, 1997, v. 27, n. 8, p. 2043, doi. 10.1002/eji.1830270831
- By:
- Publication type:
- Article
Lack of detectable defect in DNA double-strand break repair and DNA-dependent protein kinase activity in radiosensitive human severe combined immunodeficiency fibroblasts.
- Published in:
- European Journal of Immunology, 1996, v. 26, n. 5, p. 1118, doi. 10.1002/eji.1830260524
- By:
- Publication type:
- Article
Characteristics of antigen-independent and antigen-dependent interaction of dendritic cells with CD4.
- Published in:
- European Journal of Immunology, 1995, v. 25, n. 8, p. 2285, doi. 10.1002/eji.1830250826
- By:
- Publication type:
- Article
Prevention of EBV-induced B-lymphoproliferative disorder by ex vivo marrow B-cell depletion in HLA-phenoidentical or non-identical T-depleted bone marrow transplantation.
- Published in:
- British Journal of Haematology, 1998, v. 103, n. 2, p. 543, doi. 10.1046/j.1365-2141.1998.00972.x
- By:
- Publication type:
- Article
Combinatorial RNA interference as a gene therapy strategy for HIV-1 infection.
- Published in:
- 2013
- By:
- Publication type:
- Abstract