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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
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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- Publication type:
- Article
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 2, p. 266, doi. 10.1038/mt.2013.4
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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
Ex vivo expansion of umbilical cord blood (UCB) CD34<sup>+</sup> cells alters the expression and function of α4β1 and α5β1 integrins.
- Published in:
- British Journal of Haematology, 2001, v. 115, n. 1, p. 213, doi. 10.1046/j.1365-2141.2001.03084.x
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- Publication type:
- Article
Ex vivo expansion and selection of retrovirally transduced bone marrow: an efficient methodology for gene-transfer to murine lympho-haemopoietic stem cells.
- Published in:
- British Journal of Haematology, 1994, v. 87, n. 1, p. 6, doi. 10.1111/j.1365-2141.1994.tb04863.x
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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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- Publication type:
- 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
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
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
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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- 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
Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 6, p. 1083, doi. 10.1038/mt.2009.26
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- Publication type:
- Article
Characteristics of Lentiviral Vectors Harboring the Proximal Promoter of the vav Proto-oncogene: A Weak and Efficient Promoter for Gene Therapy.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 8, p. 1487, doi. 10.1038/sj.mt.6300213
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- Publication type:
- Article
Hematopoietic Dysfunction in a Mouse Model for Fanconi Anemia Group D1.
- Published in:
- Molecular Therapy, 2006, v. 14, n. 4, p. 525, doi. 10.1016/j.ymthe.2006.05.018
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- Publication type:
- Article
236. Bone Marrow-Derived Cells Do Not Contribute to Endocrine Pancreas Regeneration in Diabetic RIP/IGF-I Transgenic Mice
- Published in:
- 2005
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- Publication type:
- Abstract
Adipose-derived mesenchymal stromal cells modulate experimental autoimmune arthritis by inducing an early regulatory innate cell signature.
- Published in:
- Immunity, Inflammation & Disease, 2016, v. 4, n. 2, p. 213, doi. 10.1002/iid3.106
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- Publication type:
- 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
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
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
Mechanism of allosteric activation of human mRNA cap methyltransferase (RNMT) by RAM: insights from accelerated molecular dynamics simulations.
- Published in:
- Nucleic Acids Research, 2019, v. 47, n. 16, p. 8675, doi. 10.1093/nar/gkz613
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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
BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice.
- Published in:
- Journal of Pathology, 2015, v. 236, n. 2, p. 219, doi. 10.1002/path.4513
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- Publication type:
- 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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- Publication type:
- 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
Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10.
- Published in:
- 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.
- Published in:
- 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.
- Published in:
- 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
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
Differential sensitivity to hyperthermia of mouse normal haemopoietic stem cells related to proliferation activity and organ source.
- Published in:
- International Journal of Hyperthermia, 1987, v. 3, n. 4, p. 365, doi. 10.3109/02656738709140408
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- Publication type:
- Article
Therapeutic gene editing in CD34<sup>+</sup> hematopoietic progenitors from Fanconi anemia patients.
- Published in:
- EMBO Molecular Medicine, 2017, v. 9, n. 11, p. 1574, doi. 10.15252/emmm.201707540
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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
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
Instability of aquaglyceroporin (AQP) 2 contributes to drug resistance in Trypanosoma brucei.
- Published in:
- PLoS Neglected Tropical Diseases, 2020, v. 14, n. 7, p. 1, doi. 10.1371/journal.pntd.0008458
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- Publication type:
- Article
Cell Therapy With Mesenchymal Stem Cells Induces an Innate Immune Memory Response That Attenuates Experimental Colitis in the Long Term.
- Published in:
- Journal of Crohn's & Colitis, 2020, v. 14, n. 10, p. 1424, doi. 10.1093/ecco-jcc/jjaa079
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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
Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.
- Published in:
- PLoS ONE, 2019, v. 14, n. 10, p. 1, doi. 10.1371/journal.pone.0223775
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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>.
- Published in:
- 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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- Publication type:
- 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.
- Published in:
- 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.
- Published in:
- 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
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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- Publication type:
- Article