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Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity.
- Published in:
- EMBO Molecular Medicine, 2022, v. 14, n. 1, p. 1, doi. 10.15252/emmm.202113968
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- Publication type:
- Article
A single epidermal stem cell strategy for safe ex vivo gene therapy.
- Published in:
- EMBO Molecular Medicine, 2015, v. 7, n. 4, p. 380, doi. 10.15252/emmm.201404353
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- Publication type:
- Article
Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.
- Published in:
- EMBO Molecular Medicine, 2010, v. 2, n. 8, p. 315, doi. 10.1002/emmm.201000083
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- Publication type:
- Article
Prep1, a novel functional partner of Pbx proteins.
- Published in:
- EMBO Journal, 1998, v. 17, n. 5, p. 1423, doi. 10.1093/emboj/17.5.1423
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- Publication type:
- Article
The novel homeoprotein Prep1 modulates Pbx-Hox protein cooperativity.
- Published in:
- EMBO Journal, 1998, v. 17, n. 5, p. 1434, doi. 10.1093/emboj/17.5.1434
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- Publication type:
- Article
Functional dissection of a transcriptionally active, target-specific Hox-Pbx complex.
- Published in:
- EMBO Journal, 1997, v. 16, n. 12, p. 3644, doi. 10.1093/emboj/16.12.3644
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- Publication type:
- Article
Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts.
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- Journal of Clinical Investigation, 2012, v. 122, n. 5, p. 1653, doi. 10.1172/JCI61852
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- Publication type:
- Article
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.
- Published in:
- 2007
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- Publication type:
- journal article
Posttranscriptional control of human homeobox gene expression in induced NTERA-2 embryonal carcinoma cells.
- Published in:
- Molecular Reproduction & Development, 1989, v. 1, n. 2, p. 107, doi. 10.1002/mrd.1080010205
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- Publication type:
- Article
Gene therapy: back on track?
- Published in:
- EMBO Reports, 2010, v. 11, n. 2, p. 75, doi. 10.1038/embor.2009.284
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- Publication type:
- Article
Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology.
- Published in:
- EMBO Reports, 2008, v. 9, p. S64, doi. 10.1038/embor.2008.81
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- Publication type:
- Article
Role of CD34 Antigen in Myeloid Differentiation of Human Hematopoietic Progenitor Cells.
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- Stem Cells, 2008, v. 26, n. 4, p. 950, doi. 10.1634/stemcells.2007-0597
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- Publication type:
- Article
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.
- Published in:
- Nature Medicine, 2006, v. 12, n. 12, p. 1397, doi. 10.1038/nm1504
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- Publication type:
- Article
Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.
- Published in:
- Scientific Reports, 2016, p. 24724, doi. 10.1038/srep24724
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- Publication type:
- Article
Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.
- Published in:
- PLoS ONE, 2015, v. 10, n. 5, p. 1, doi. 10.1371/journal.pone.0126590
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- Publication type:
- Article
Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells.
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- PLoS ONE, 2014, v. 9, n. 11, p. 1, doi. 10.1371/journal.pone.0112712
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- Publication type:
- Article
Deletion of the LTR Enhancer/Promoter Has No Impact on the Integration Profile of MLV Vectors in Human Hematopoietic Progenitors.
- Published in:
- PLoS ONE, 2013, v. 8, n. 1, p. 1, doi. 10.1371/journal.pone.0055721
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- Publication type:
- Article
The GATA1-HS2 Enhancer Allows Persistent and Position- Independent Expression of a β-globin Transgene.
- Published in:
- PLoS ONE, 2011, v. 6, n. 12, p. 1, doi. 10.1371/journal.pone.0027955
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- Publication type:
- Article
High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells After Donor Lymphocyte Infusion.
- Published in:
- PLoS ONE, 2010, v. 5, n. 12, p. 1, doi. 10.1371/journal.pone.0015688
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- Publication type:
- Article
Transcription Factor Binding Sites Are Genetic Determinants of Retroviral Integration in the Human Genome.
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- PLoS ONE, 2009, v. 4, n. 2, p. 1, doi. 10.1371/journal.pone.0004571
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- Publication type:
- Article
Genotoxic Signature in Cord Blood Cells of Newborns Exposed In Utero to a Zidovudine-Based Antiretroviral Combination.
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- Journal of Infectious Diseases, 2013, v. 208, n. 2, p. 235, doi. 10.1093/infdis/jit149
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- Publication type:
- Article
Differential and stage-related expression in embryonic tissues of a new human homoeobox gene.
- Published in:
- Nature, 1986, v. 324, n. 6098, p. 664, doi. 10.1038/324664a0
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- Publication type:
- Article
Repairing Without Cutting: A Safer Alternative to Gene Correction?
- Published in:
- Molecular Therapy, 2014, v. 22, n. 4, p. 690, doi. 10.1038/mt.2014.40
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- Publication type:
- Article
Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 9, p. 1695, doi. 10.1038/mt.2013.143
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- Publication type:
- Article
Genome-wide analysis of promoter and enhancer usage in hematopoietic stem cell differentiation.
- Published in:
- 2013
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- Publication type:
- Conference Paper/Materials
Correction: Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells.
- Published in:
- 2020
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- Publication type:
- Correction Notice
Estimated Comparative Integration Hotspots Identify Different Behaviors of Retroviral Gene Transfer Vectors.
- Published in:
- PLoS Computational Biology, 2011, v. 7, n. 12, p. 1, doi. 10.1371/journal.pcbi.1002292
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- Publication type:
- Article
PPARδ is a ligand-dependent negative regulator of vitamin D3-induced monocyte differentiation.
- Published in:
- Carcinogenesis, 2009, v. 30, n. 2, p. 230, doi. 10.1093/carcin/bgn272
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- Publication type:
- Article
Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases.
- Published in:
- Viruses (1999-4915), 2021, v. 13, n. 8, p. 1526, doi. 10.3390/v13081526
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- Publication type:
- Article
The future of gene therapy.
- Published in:
- 2004
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- Publication type:
- Book Review
Failure to correct murine muscular dystrophy.
- Published in:
- Nature, 2001, v. 411, n. 6841, p. 1014, doi. 10.1038/35082631
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- Publication type:
- Article
C/EBPδ regulates cell cycle and self-renewal of human limbal stem cells.
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- Journal of Cell Biology, 2007, v. 177, n. 6, p. 1037, doi. 10.1083/jcb.200703003
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- Publication type:
- Article
Nuclear architecture dictates HIV-1 integration site selection.
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- Nature, 2015, v. 521, n. 7551, p. 227, doi. 10.1038/nature14226
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- Publication type:
- Article
Gene therapies need new development models.
- Published in:
- Nature, 2012, v. 490, n. 7418, p. 7, doi. 10.1038/490007a
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- Publication type:
- Article
Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 4, p. 798, doi. 10.1038/mt.2011.233
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- 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
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
Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 5, p. 851, doi. 10.1038/mt.2009.51
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- Publication type:
- Article
Correction of Laminin-5 Deficiency in Human Epidermal Stem Cells by Transcriptionally Targeted Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 12, p. 1977, doi. 10.1038/mt.2008.204
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- Publication type:
- Article
Long-term Engraftment of Single Genetically Modified Human Epidermal Holoclones Enables Safety Pre-assessment of Cutaneous Gene Therapy.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 9, p. 1670, doi. 10.1038/sj.mt.6300238
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- Publication type:
- Article
606. Identification of a 45-aa Domain of the F12-Vif Mutant Possessing Anti-HIV Activity.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S234, doi. 10.1016/j.ymthe.2006.08.680
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- Publication type:
- Article
725. Correction of Laminin-5-Deficient Junctional Epidermolysis Bullosa by Transplantation of Genetically Modified Epidermal Stem Cells. A Phase-I Clinical Trial.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S280, doi. 10.1016/j.ymthe.2006.08.805
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- Publication type:
- Article
734. The Influence of Vector Design on Integration Site Selection by gamma-Retroviral and Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S284, doi. 10.1016/j.ymthe.2006.08.815
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- Publication type:
- Article
811. Correction of Laminin-5 β3 Chain Deficiency in Human Epidermal Stem Cells by Transcriptionally Targeted Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S314, doi. 10.1016/j.ymthe.2006.08.896
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- Publication type:
- Article
826. Transduction of Human Hematopoietic Stem Cells by RD114-TR-Pseudotyped Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S320, doi. 10.1016/j.ymthe.2006.08.911
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- Publication type:
- Article
T Lymphocytes Transduced with a Lentiviral Vector Expressing F12-vif Are Protected from HIV-1 Infection in an APOBEC3G-Independent Manner
- Published in:
- Molecular Therapy, 2005, v. 12, n. 4, p. 697, doi. 10.1016/j.ymthe.2005.05.014
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- Publication type:
- Article
819. Molecular Follow-Up of Patients Treated with Allogeneic Hematopoietic Stem Cell Transplantation and Donor Lymphocytes Transduced with a Retroviral Vector Expressing HSV-TK and ÄLNGFR
- Published in:
- 2005
- By:
- Publication type:
- Abstract
64. Characterization of the Mechanism of Action of the Anti-HIV-1 Transgene F12-Vif
- Published in:
- 2005
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- Publication type:
- Abstract
Site-Specific Integration of Functional Transgenes into the Human Genome by Adeno/AAV Hybrid Vectors
- Published in:
- Molecular Therapy, 2004, v. 10, n. 4, p. 660, doi. 10.1016/j.ymthe.2004.07.003
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- Publication type:
- Article
Characterization of Two Neuroblastoma Cell Lines Expressing Recombinant Nerve Growth Factor Receptors.
- Published in:
- Journal of Neurochemistry, 1991, v. 56, n. 1, p. 67, doi. 10.1111/j.1471-4159.1991.tb02563.x
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- Publication type:
- Article