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Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside.
- Published in:
- Cells (2073-4409), 2021, v. 10, n. 12, p. 3273, doi. 10.3390/cells10123273
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- Publication type:
- Article
Gene editing improves endoplasmic reticulum-mitochondrial contacts and unfolded protein response in Friedreich’s ataxia iPSC-derived neurons.
- Published in:
- Frontiers in Pharmacology, 2024, p. 1, doi. 10.3389/fphar.2024.1323491
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- Publication type:
- Article
Potential use of stem cells as a therapy for cystinosis.
- Published in:
- Pediatric Nephrology, 2019, v. 34, n. 6, p. 965, doi. 10.1007/s00467-018-3974-7
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- Publication type:
- Article
Kidney repair and stem cells: a complex and controversial process.
- Published in:
- Pediatric Nephrology, 2011, v. 26, n. 9, p. 1427, doi. 10.1007/s00467-011-1789-x
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- Publication type:
- Article
Chaperone‐mediated autophagy is impaired in the lysosomal storage disorder cystinosis (740.2).
- Published in:
- FASEB Journal, 2014, v. 28, p. N.PAG, doi. 10.1096/fasebj.28.1_supplement.740.2
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- Publication type:
- Article
Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 2, p. 433, doi. 10.1038/mt.2012.214
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- Publication type:
- Article
Molecular pathogenesis of cystinosis: effect of CTNS mutations on the transport activity and subcellular localization of cystinosin.
- Published in:
- Human Molecular Genetics, 2004, v. 13, n. 13, p. 1361, doi. 10.1093/hmg/ddh152
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- Publication type:
- Article
Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia.
- Published in:
- Science Translational Medicine, 2017, v. 9, n. 413, p. 1, doi. 10.1126/scitranslmed.aaj2347
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- Publication type:
- Article
Identification and Characterisation of the Murine Homologue of the Gene Responsible for Cystinosis, Ctns.
- Published in:
- BMC Genomics, 2000, v. 1, p. 1
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- Publication type:
- Article
Impairment of chaperone-mediated autophagy leads to selective lysosomal degradation defects in the lysosomal storage disease cystinosis.
- Published in:
- EMBO Molecular Medicine, 2015, v. 7, n. 2, p. 158, doi. 10.15252/emmm.201404223
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- Publication type:
- Article
Nephropathic cystinosis: an international consensus document.
- Published in:
- Nephrology Dialysis Transplantation, 2014, v. 29, n. suppl_4, p. iv87, doi. 10.1093/ndt/gfu090
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- Publication type:
- Article
Is genetic rescue of cystinosis an achievable treatment goal?
- Published in:
- Nephrology Dialysis Transplantation, 2014, v. 29, n. 3, p. 522, doi. 10.1093/ndt/gft270
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- Publication type:
- Article
Lentiviral Gene Delivery of vMIP-II to Transplanted Endothelial Cells and Endothelial Progenitors Is Proangiogenic In Vivo.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 7, p. 1264, doi. 10.1038/sj.mt.6300183
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- Publication type:
- Article
26. Lentiviral Vector Delivery of vMIP-II Using Both Transplanted Endothelial and Progenitor Cells Enhances a Robust and Functional Angiogenesis In Vivo.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S11, doi. 10.1016/j.ymthe.2006.08.038
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- Publication type:
- Article
Hematopoietic Stem Cell Gene Therapy for Brain Metastases Using Myeloid Cell-Specific Gene Promoters.
- Published in:
- 2020
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- Publication type:
- journal article
Muscle wasting and adipose tissue browning in infantile nephropathic cystinosis.
- Published in:
- Journal of Cachexia, Sarcopenia & Muscle, 2016, v. 7, n. 2, p. 152, doi. 10.1002/jcsm.12056
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- Publication type:
- Article
Macrophage polarization impacts tunneling nanotube formation and intercellular organelle trafficking.
- Published in:
- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-50971-x
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- Publication type:
- Article
Cysteamine therapy: a treatment for cystinosis, not a cure.
- Published in:
- Kidney International, 2012, v. 81, n. 2, p. 127, doi. 10.1038/ki.2011.301
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- Publication type:
- Article
Kidney preservation by bone marrow cell transplantation in hereditary nephropathy.
- Published in:
- Kidney International, 2011, v. 79, n. 11, p. 1198, doi. 10.1038/ki.2010.537
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- Publication type:
- Article
Brief Reports: Lysosomal Cross-Correction by Hematopoietic Stem Cell-Derived Macrophages Via Tunneling Nanotubes.
- Published in:
- Stem Cells, 2015, v. 33, n. 1, p. 301, doi. 10.1002/stem.1835
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- Publication type:
- Article
Non-invasive intradermal imaging of cystine crystals in cystinosis.
- Published in:
- PLoS ONE, 2021, v. 16, n. 3, p. 1, doi. 10.1371/journal.pone.0247846
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- Publication type:
- Article
Cystinosin, the protein defective in cystinosis, is a H<sup>+</sup>-driven lysosomal cystine transporter.
- Published in:
- EMBO Journal, 2001, v. 20, n. 21, p. 5940, doi. 10.1093/emboj/20.21.5940
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- Publication type:
- Article
Cystinose: De la découverte du gène aux premiers essais de thérapie génique.
- Published in:
- Médecine Sciences, 2023, v. 39, n. 3, p. 253, doi. 10.1051/medsci/2023025
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- Publication type:
- Article
Severity of phenotype in cystinosis varies with mutations in the CTNS gene: predicted effect on the model of cystinosin.
- Published in:
- Human Molecular Genetics, 1999, v. 8, n. 13, p. 2507, doi. 10.1093/hmg/8.13.2507
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- Publication type:
- Article
Tunneling Nanotubes and Gap Junctions-Their Role in Long-Range Intercellular Communication during Development, Health, and Disease Conditions.
- Published in:
- Frontiers in Molecular Neuroscience, 2017, p. 1, doi. 10.3389/fnmol.2017.00333
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- Publication type:
- Article