Found: 19
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An AAVS1-Targeted Minigene Platform for Correction of iPSCs From All Five Types of Chronic Granulomatous Disease.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 1, p. 147, doi. 10.1038/mt.2014.195
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- Publication type:
- Article
False-Positive HIV PCR Test Following Ex Vivo Lentiviral Gene Transfer Treatment of X-linked Severe Combined Immunodeficiency Vector.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 2, p. 244, doi. 10.1038/mt.2013.296
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- Publication type:
- Article
CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.
- Published in:
- Science Translational Medicine, 2017, v. 9, n. 372, p. 1, doi. 10.1126/scitranslmed.aah3480
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- Publication type:
- Article
Geosmithia argillacea: An Emerging Cause of Invasive Mycosis in Human Chronic Granulomatous Disease.
- Published in:
- Clinical Infectious Diseases, 2011, v. 52, n. 6, p. e136, doi. 10.1093/cid/ciq250
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- Publication type:
- Article
Mobilization characteristics and strategies to improve hematopoietic progenitor cell mobilization and collection in patients with chronic granulomatous disease and severe combined immunodeficiency.
- Published in:
- Transfusion, 2015, v. 55, n. 2, p. 265, doi. 10.1111/trf.12830
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- Publication type:
- Article
468. Evaluation of Factors Influencing the Production Titers of SIV-Derived Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S181, doi. 10.1016/j.ymthe.2006.08.537
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- Publication type:
- Article
671. SIV-Derived Lentiviral Vectors Pseudotyped with Modified RD114 Envelope and Containing Mutant MGMT Efficiently Transduce Human CD34+ PBSCs and Allow Selection.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S259, doi. 10.1016/j.ymthe.2006.08.748
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- Publication type:
- Article
806. Immunologic Improvement after Gene Therapy in 3 Preadolescents with X-Linked Severe Combined Immunodeficiency (XSCID).
- Published in:
- Molecular Therapy, 2006, v. 13, p. S312, doi. 10.1016/j.ymthe.2006.08.1227
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- Publication type:
- Article
Short Stature in Partially Corrected X-linked Severe Combined Immunodeficiency - Suboptimal Response to Growth Hormone.
- Published in:
- Journal of Pediatric Endocrinology & Metabolism, 2008, v. 21, n. 11, p. 1057, doi. 10.1515/JPEM.2008.21.11.1057
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- Publication type:
- Article
Progressive B Cell Loss in Revertant X-SCID.
- Published in:
- Journal of Clinical Immunology, 2020, v. 40, n. 7, p. 1001, doi. 10.1007/s10875-020-00825-3
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- Publication type:
- Article
Targeted gene addition in human CD34<sup>+</sup> hematopoietic cells for correction of X-linked chronic granulomatous disease.
- Published in:
- Nature Biotechnology, 2016, v. 34, n. 4, p. 424, doi. 10.1038/nbt.3513
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- Publication type:
- Article
Interleukin-5 does not influence differential transcription of transmembrane and soluble isoforms of IL-5R α in vivo.
- Published in:
- European Journal of Haematology, 2006, v. 77, n. 3, p. 181, doi. 10.1111/j.1600-0609.2006.00699.x
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- Publication type:
- Article
Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease‐associated inflammatory bowel disease.
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- Journal of Gastroenterology & Hepatology, 2023, v. 38, n. 12, p. 2083, doi. 10.1111/jgh.16342
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- Publication type:
- Article
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-31344-x
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- Publication type:
- Article
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.
- Published in:
- Science Translational Medicine, 2016, v. 8, n. 335, p. 1, doi. 10.1126/scitranslmed.aad8856
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- Publication type:
- Article
Test Dose Pharmacokinetics in Pediatric Patients Receiving Once‐Daily IV Busulfan Conditioning for Hematopoietic Stem Cell Transplant: A Reliable Approach?
- Published in:
- Journal of Clinical Pharmacology, 2018, v. 58, n. 3, p. 332, doi. 10.1002/jcph.1049
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- Publication type:
- Article
Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency.
- Published in:
- 2016
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- Publication type:
- journal article
Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency.
- Published in:
- 2015
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- Publication type:
- journal article
Severe combined immunodeficiency: A national surveillance study.
- Published in:
- Pediatric Allergy & Immunology, 2008, v. 19, n. 4, p. 298, doi. 10.1111/j.1399-3038.2007.00646.x
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- Publication type:
- Article