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Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-34479-z
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- Publication type:
- Article
Therapeutic gene editing in CD34<sup>+</sup> hematopoietic progenitors from Fanconi anemia patients.
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- EMBO Molecular Medicine, 2017, v. 9, n. 11, p. 1574, doi. 10.15252/emmm.201707540
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- Article
Fanconi anaemia: from a monogenic disease to sporadic cancer.
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- Clinical & Translational Oncology, 2011, v. 13, n. 4, p. 215, doi. 10.1007/s12094-011-0645-6
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- Article
BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice.
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- Journal of Pathology, 2015, v. 236, n. 2, p. 219, doi. 10.1002/path.4513
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- Publication type:
- Article
Cytotoxic Infection of Hematopoietic Stem and Committed Progenitor Cells by the Parvovirus Minute Virus of Mice Propagation of an Acute Myelosuppression in Culture<sup>a</sup>.
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- Annals of the New York Academy of Sciences, 1991, v. 628, n. 1, p. 262, doi. 10.1111/j.1749-6632.1991.tb17255.x
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- Article
Molecular basis of RNA guanine-7 methyltransferase (RNMT) activation by RAM.
- Published in:
- Nucleic Acids Research, 2016, v. 44, n. 21, p. 10423, doi. 10.1093/nar/gkw637
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- Publication type:
- Article
Temperature-induced melting of double-stranded DNA in the absence and presence of covalently bonded antitumour drugs: insight from molecular dynamics simulations.
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- Nucleic Acids Research, 2011, v. 39, n. 18, p. 8248, doi. 10.1093/nar/gkr512
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- Publication type:
- Article
Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse transplantation model.
- Published in:
- Stem Cell Research & Therapy, 2015, v. 6, n. 1, p. 1, doi. 10.1186/s13287-015-0155-5
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- Publication type:
- Article
Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse transplantation model.
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- Stem Cell Research & Therapy, 2015, v. 6, n. 1, p. 165, doi. 10.1186/s13287-015-0155-5
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- Publication type:
- Article
Human Adipose-Derived Mesenchymal Stem Cells Modulate Experimental Autoimmune Arthritis by Modifying Early Adaptive T Cell Responses.
- Published in:
- Stem Cells, 2015, v. 33, n. 12, p. 3493, doi. 10.1002/stem.2113
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- Article
Conversion of Human Fibroblasts Into Monocyte-Like Progenitor Cells.
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- Stem Cells, 2014, v. 32, n. 11, p. 2923, doi. 10.1002/stem.1800
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- Article
Brief Report: Reduced Expression of CD18 Leads to the In Vivo Expansion of Hematopoietic Stem Cells in Mouse Bone Marrow.
- Published in:
- Stem Cells, 2014, v. 32, n. 10, p. 2794, doi. 10.1002/stem.1762
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- Article
Brief Report: Impaired Cell Reprogramming in Nonhomologous End Joining Deficient Cells.
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- Stem Cells, 2013, v. 31, n. 8, p. 1726, doi. 10.1002/stem.1406
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- Publication type:
- Article
Adipose Tissue-Derived Mesenchymal Stem Cells Have In Vivo Immunosuppressive Properties Applicable for the Control of the Graft-Versus-Host Disease.
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- Stem Cells, 2006, v. 24, n. 11, p. 2582, doi. 10.1634/stemcells.2006-0228
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- Publication type:
- Article
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231188979
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- Publication type:
- Article
Long-term skin regeneration in xenografts from iPSC teratoma-derived human keratinocytes.
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- Experimental Dermatology, 2016, v. 25, n. 9, p. 736, doi. 10.1111/exd.13049
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- Publication type:
- Article
In vivo imaging of lung inflammation with neutrophil-specific <sup>68</sup>Ga nano-radiotracer.
- Published in:
- Scientific Reports, 2017, v. 7, n. 1, p. 1, doi. 10.1038/s41598-017-12829-y
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- Publication type:
- Article
Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10.
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- Stem Cell Research & Therapy, 2021, v. 12, n. 1, p. 1, doi. 10.1186/s13287-021-02193-0
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- Publication type:
- Article
Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.
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- Stem Cell Research & Therapy, 2021, v. 12, n. 1, p. 1, doi. 10.1186/s13287-021-02145-8
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- Publication type:
- Article
Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.
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- Stem Cell Research & Therapy, 2021, v. 12, n. 1, p. 1, doi. 10.1186/s13287-021-02145-8
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- Publication type:
- Article
Epigenetic Alterations in Fanconi Anaemia: Role in Pathophysiology and Therapeutic Potential.
- Published in:
- PLoS ONE, 2015, v. 10, n. 10, p. 1, doi. 10.1371/journal.pone.0139740
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- Publication type:
- Article
Transforming and Tumorigenic Activity of JAK2 by Fusion to BCR: Molecular Mechanisms of Action of a Novel BCR-JAK2 Tyrosine-Kinase.
- Published in:
- PLoS ONE, 2012, v. 7, n. 2, p. 1, doi. 10.1371/journal.pone.0032451
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- Publication type:
- Article
Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using Induced Pluripotent Stem Cells.
- Published in:
- PLoS ONE, 2011, v. 6, n. 3, p. 1, doi. 10.1371/journal.pone.0017565
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- Publication type:
- Article
Bcr/Abl Interferes with the Fanconi Anemia/BRCA Pathway: Implications in the Chromosomal Instability of Chronic Myeloid Leukemia Cells.
- Published in:
- PLoS ONE, 2010, v. 5, n. 12, p. 1, doi. 10.1371/journal.pone.0015525
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- Publication type:
- Article
Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization.
- Published in:
- 2020
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- Publication type:
- journal article
Mechanistic Insight into the Catalytic Activity of ββα-Metallonucleases from Computer Simulations: Vibrio vulnificus Periplasmic Nuclease as a Test Case.
- Published in:
- ChemBioChem, 2011, v. 12, n. 17, p. 2615, doi. 10.1002/cbic.201100485
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- Publication type:
- Article
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.
- Published in:
- Nature, 2009, v. 460, n. 7251, p. 53, doi. 10.1038/nature08129
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- Article
Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 3, p. 482, doi. 10.1038/mt.2014.11
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- Article
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases.
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- Molecular Therapy, 2013, v. 21, n. 2, p. 266, doi. 10.1038/mt.2013.4
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- Article
TALEN mediated gene editing in a mouse model of Fanconi anemia.
- Published in:
- Scientific Reports, 2020, v. 10, n. 1, p. 1, doi. 10.1038/s41598-020-63971-z
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- Publication type:
- Article
Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model.
- Published in:
- Frontiers in Immunology, 2023, v. 14, p. 1, doi. 10.3389/fimmu.2023.1062086
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- Publication type:
- Article
Instability of aquaglyceroporin (AQP) 2 contributes to drug resistance in Trypanosoma brucei.
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- PLoS Neglected Tropical Diseases, 2020, v. 14, n. 7, p. 1, doi. 10.1371/journal.pntd.0008458
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- Article
Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.
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- PLoS ONE, 2019, v. 14, n. 10, p. 1, doi. 10.1371/journal.pone.0223775
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- Publication type:
- Article
Elucidation of Ligand-Dependent Modulation of Disorder-Order Transitions in the Oncoprotein MDM2.
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- PLoS Computational Biology, 2015, v. 11, n. 6, p. 1, doi. 10.1371/journal.pcbi.1004282
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- Article
Tumor cells as cellular vehicles to deliver gene therapies to metastatic tumors.
- Published in:
- Cancer Gene Therapy, 2005, v. 12, n. 4, p. 341, doi. 10.1038/sj.cgt.7700801
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- Article
Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis.
- Published in:
- Frontiers in Immunology, 2021, v. 12, p. 1, doi. 10.3389/fimmu.2021.687443
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- Publication type:
- Article
CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative.
- Published in:
- Clinical Genetics, 2022, v. 101, n. 5/6, p. 481, doi. 10.1111/cge.14113
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- Article
Advances in the gene therapy of monogenic blood cell diseases.
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- Clinical Genetics, 2020, v. 97, n. 1, p. 89, doi. 10.1111/cge.13593
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- Article
Adipose-derived mesenchymal stromal cells modulate experimental autoimmune arthritis by inducing an early regulatory innate cell signature.
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- Immunity, Inflammation & Disease, 2016, v. 4, n. 2, p. 213, doi. 10.1002/iid3.106
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- Publication type:
- Article
Biodistribution and Efficacy of Human Adipose-Derived Mesenchymal Stem Cells Following Intranodal Administration in Experimental Colitis.
- Published in:
- Frontiers in Immunology, 2017, p. 1, doi. 10.3389/fimmu.2017.00638
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- Publication type:
- Article
Intralymphatic Administration of Adipose Mesenchymal Stem Cells Reduces the Severity of Collagen-Induced Experimental Arthritis.
- Published in:
- Frontiers in Immunology, 2017, v. 8, p. 1, doi. 10.3389/fimmu.2017.00462
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- Publication type:
- Article
Intralymphatic Administration of Adipose Mesenchymal Stem Cells Reduces the Severity of Collagen-Induced Experimental Arthritis.
- Published in:
- Frontiers in Immunology, 2017, v. 8, p. 1, doi. 10.3389/fimmu.2017.00462
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- Publication type:
- Article
The downregulated membrane expression of CD18 in CD34+ cells defines a primitive population of human hematopoietic stem cells.
- Published in:
- Stem Cell Research & Therapy, 2020, v. 11, n. 1, p. 1, doi. 10.1186/s13287-020-01672-0
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- Publication type:
- Article
Endocrine Pancreas Regeneration in Diabetic IGF-I Transgenic Mice Results from Replication of Pre-Existing β-Cells Rather than from Bone Marrow Cell Recruitment and Differentiation.
- Published in:
- Diabetes, 2007, v. 56, p. A424
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- Publication type:
- Article
Lentiviral vector integration sites in human NOD/SCID repopulating cells.
- Published in:
- Journal of Gene Medicine, 2006, v. 8, n. 10, p. 1197, doi. 10.1002/jgm.958
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- Article
Systematic analysis of clinically applicable conditions leading to a high efficiency of transduction and transgene expression in human T cells.
- Published in:
- Journal of Gene Medicine, 2001, v. 3, n. 1, p. 32, doi. 10.1002/1521-2254(2000)9999:9999<::AID-JGM153>3.0.CO;2-R
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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
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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
In Vivo Delivery of Antigens by Adenovirus Dodecahedron Induces Cellular and Humoral Immune Responses to Elicit Antitumor Immunity.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 5, p. 1046, doi. 10.1038/mt.2010.16
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- Publication type:
- Article
Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 12, p. 2000, doi. 10.1038/mt.2009.200
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- Article