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Development of anti-PAX3 immune responses; a target for cancer immunotherapy.
- Published in:
- Cancer Immunology, Immunotherapy, 2007, v. 56, n. 9, p. 1381, doi. 10.1007/s00262-007-0294-3
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- Publication type:
- Article
WASP: a key immunological multitasker.
- Published in:
- 2010
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- Publication type:
- journal article
Wasp in immune-system organization and function.
- Published in:
- Nature Reviews Immunology, 2002, v. 2, n. 9, p. 635, doi. 10.1038/nri884
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- Publication type:
- Article
Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy.
- Published in:
- Scientific Reports, 2016, p. 19750, doi. 10.1038/srep19750
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- Publication type:
- Article
Construction of stable packaging cell lines for clinical lentiviral vector production.
- Published in:
- Scientific Reports, 2015, p. 9021, doi. 10.1038/srep09021
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- Publication type:
- Article
Efficient generation of transgenic mice by lentivirus-mediated modification of spermatozoa.
- Published in:
- FASEB Journal, 2014, v. 28, n. 2, p. 569, doi. 10.1096/fj.13-233999
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- Publication type:
- Article
Tailoring cells for clinical needs: Meeting report from the Advanced Therapy in Healthcare symposium (October 28-29 2017, Doha, Qatar).
- Published in:
- 2018
- By:
- Publication type:
- research
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
Gene therapy: Is IL2RG oncogenic in T-cell development?
- Published in:
- Nature, 2006, v. 443, n. 7109, p. E5, doi. 10.1038/nature05218
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- Publication type:
- Article
The future of gene therapy.
- Published in:
- 2004
- By:
- Publication type:
- Book Review
Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 4, p. 737, doi. 10.1038/mt.2014.242
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- Publication type:
- Article
'Darwinian' Tumor-Suppression Model Unsupported in Clinical Experience.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 9, p. 1562, doi. 10.1038/mt.2014.147
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- Publication type:
- Article
Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 3, p. 607, doi. 10.1038/mt.2013.265
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- Publication type:
- Article
Moving Forward on Shifting Sands: Ethical Regulation of Gene Therapy Clinical Trials in the United Kingdom.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 4, p. 715, doi. 10.1038/mt.2013.43
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- Publication type:
- Article
'Special Exemptions': Should They Be Put on Trial?
- Published in:
- Molecular Therapy, 2013, v. 21, n. 2, p. 261, doi. 10.1038/mt.2013.1
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- Publication type:
- Article
Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells.
- Published in:
- Human Molecular Genetics, 2011, v. 20, n. 15, p. 2905, doi. 10.1093/hmg/ddr144
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- Publication type:
- Article
Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells.
- Published in:
- Nature Communications, 2017, v. 8, n. 1, p. 1, doi. 10.1038/s41467-017-01676-0
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- Publication type:
- Article
Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection.
- Published in:
- Nature Communications, 2016, v. 7, n. 12, p. 13992, doi. 10.1038/ncomms13992
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- Publication type:
- Article
Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.
- Published in:
- Nature Communications, 2016, v. 7, n. 7, p. 12175, doi. 10.1038/ncomms12175
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- Publication type:
- Article
Platelet actin nodules are podosome-like structures dependent on Wiskott-Aldrich syndrome protein and ARP2/3 complex.
- Published in:
- Nature Communications, 2015, v. 6, n. 6, p. 7254, doi. 10.1038/ncomms8254
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- Publication type:
- Article
Restoring the natural tropism of AAV2 vectors for human liver.
- Published in:
- Science Translational Medicine, 2020, v. 12, n. 560, p. 1, doi. 10.1126/scitranslmed.aba3312
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- Publication type:
- Article
Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cells.
- Published in:
- eLife, 2020, p. 1, doi. 10.7554/eLife.55547
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- Publication type:
- Article
Lentiviral vector transduction of spermatozoa as a tool for the study of early development.
- Published in:
- FEBS Open Bio, 2014, v. 4, p. 266, doi. 10.1016/j.fob.2014.02.008
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- Publication type:
- Article
Lentiviral vector transduction of spermatozoa as a tool for the study of early development.
- Published in:
- FEBS Open Bio, 2014, v. 4, n. 1, p. 266, doi. 10.1016/j.fob.2014.02.008
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- Publication type:
- Article
Correction: The Tight Junction Associated Signalling Proteins ZO-1 and ZONAB Regulate Retinal Pigment Epithelium Homeostasis in Mice.
- Published in:
- 2023
- By:
- Publication type:
- Correction Notice
Valproic Acid Confers Functional Pluripotency to Human Amniotic Fluid Stem Cells in a Transgene-free Approach.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 10, p. 1953, doi. 10.1038/mt.2012.117
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- Publication type:
- Article
Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 10, p. 1968, doi. 10.1038/mt.2012.110
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- Publication type:
- Article
The β-Globin Locus Control Region in Combination With the EF1α Short Promoter Allows Enhanced Lentiviral Vector-mediated Erythroid Gene Expression With Conserved Multilineage Activity.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 7, p. 1400, doi. 10.1038/mt.2012.50
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- Publication type:
- Article
Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 10, p. 1867, doi. 10.1038/mt.2011.127
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- Publication type:
- Article
iPSCs: Unstable Origins?
- Published in:
- 2011
- By:
- Publication type:
- Opinion
Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 4, p. 703, doi. 10.1038/mt.2011.19
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- Publication type:
- Article
Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 2, p. 408, doi. 10.1038/mt.2010.201
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- Publication type:
- Article
Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 1, p. 122, doi. 10.1038/mt.2010.226
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- Publication type:
- Article
Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 1, p. 28, doi. 10.1038/mt.2010.232
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- Publication type:
- Article
Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 12, p. 2139, doi. 10.1038/mt.2010.231
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- Publication type:
- Article
Transatlantic Consortium Spotlights Need for Changes in Gene Therapy Trials.
- Published in:
- 2010
- By:
- Publication type:
- Editorial
A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation–mediated Silencing of Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 9, p. 1640, doi. 10.1038/mt.2010.132
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- Publication type:
- Article
Hybrid Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 7, p. 1263, doi. 10.1038/mt.2010.76
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- 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
- By:
- Publication type:
- Article
Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 11, p. 1919, doi. 10.1038/mt.2009.179
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- Publication type:
- Article
Sleeping Beauty Transposition From Nonintegrating Lentivirus.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 7, p. 1197, doi. 10.1038/mt.2009.94
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- Publication type:
- Article
Tolerance Induction Using Lentiviral Gene Delivery Delays Onset and Severity of Collagen II Arthritis.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 4, p. 632, doi. 10.1038/mt.2009.299
- By:
- Publication type:
- Article
Improvement of Migratory Defects in a Murine Model of Wiskott–Aldrich Syndrome Gene Therapy.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 5, p. 836, doi. 10.1038/mt.2008.43
- By:
- Publication type:
- Article
Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 3, p. 590, doi. 10.1038/sj.mt.6300393
- By:
- Publication type:
- Article
Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 11, p. 1947, doi. 10.1038/sj.mt.6300281
- By:
- Publication type:
- Article
Lentiviral Vectors for T-cell Suicide Gene Therapy: Preservation of T-cell Effector Function After Cytokine-mediated Transduction.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 2, p. 355, doi. 10.1038/sj.mt.6300042
- By:
- Publication type:
- Article
Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning.
- Published in:
- Molecular Therapy, 2006, v. 14, n. 4, p. 505, doi. 10.1016/j.ymthe.2006.06.007
- By:
- Publication type:
- Article
929. Functional Potential of Human T Cells Following Lentiviral Suicide Gene Transduction.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S359, doi. 10.1016/j.ymthe.2006.08.1020
- By:
- Publication type:
- Article
65. Long-Term Effects of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome: Persistence of Functional Correction of T Cells and Lack of Malignant Trasformation.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S27, doi. 10.1016/j.ymthe.2006.08.081
- By:
- Publication type:
- Article
350. Ectopic Retroviral Expression of LMO2, but Not IL2Rγ, Blocks Human T-Cell Development from CD34+ Progenitor Cells: Implications for Gene Therapy.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S133, doi. 10.1016/j.ymthe.2006.08.408
- By:
- Publication type:
- Article