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Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison's disease improves survival and neurological outcomes.
- Published in:
- 2011
- By:
- Publication type:
- journal article
A living band-aid for epidermolysis bullosa.
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- Blood Transfusion (17232007), 2015, v. 13, n. 1, p. 1, doi. 10.2450/2014.0289-14
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- Publication type:
- Article
Gene editing and its application for hematological diseases.
- Published in:
- 2016
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- Publication type:
- journal article
Engineering Human Cells Expressing CRISPR/Cas9-Synergistic Activation Mediators for Recombinant Protein Production.
- Published in:
- International Journal of Molecular Sciences, 2023, v. 24, n. 10, p. 8468, doi. 10.3390/ijms24108468
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- Publication type:
- Article
Correction of Fanconi Anemia Mutations Using Digital Genome Engineering.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 15, p. 8416, doi. 10.3390/ijms23158416
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- Publication type:
- Article
Engineering CRISPR/Cas9 for Multiplexed Recombinant Coagulation Factor Production.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 9, p. 5090, doi. 10.3390/ijms23095090
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- Publication type:
- Article
CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression.
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- International Journal of Molecular Sciences, 2018, v. 19, n. 4, p. 946, doi. 10.3390/ijms19040946
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- Publication type:
- Article
Incorporation of Immune Checkpoint Blockade into Chimeric Antigen Receptor T Cells (CAR-Ts): Combination or Built-In CAR-T.
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- International Journal of Molecular Sciences, 2018, v. 19, n. 2, p. 340, doi. 10.3390/ijms19020340
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- Publication type:
- Article
CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells.
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- International Journal of Molecular Sciences, 2017, v. 18, n. 6, p. 1269, doi. 10.3390/ijms18061269
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- Publication type:
- Article
Rapid DNA replication origin licensing protects stem cell pluripotency.
- Published in:
- eLife, 2017, p. 1, doi. 10.7554/eLife.30473.001
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- Publication type:
- Article
Preconditioning of mesenchymal stem cells for improved transplantation efficacy in recessive dystrophic epidermolysis bullosa.
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- Stem Cell Research & Therapy, 2014, v. 5, n. 6, p. 121, doi. 10.1186/scrt511
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- Publication type:
- Article
α-L-iduronidase therapy for mucopolysaccharidosis type I.
- Published in:
- Biologics: Targets & Therapy, 2008, v. 2, n. 4, p. 743
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- Publication type:
- Article
Creation and characterization of novel rat model for recessive dystrophic epidermolysis bullosa: Frameshift mutation of the Col7a1 gene leads to severe blistered phenotype.
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- PLoS ONE, 2024, v. 19, n. 5, p. 1, doi. 10.1371/journal.pone.0302991
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- Publication type:
- Article
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity.
- Published in:
- Nature Biotechnology, 2023, v. 41, n. 5, p. 673, doi. 10.1038/s41587-022-01533-6
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- Publication type:
- Article
sdf1 Expression Reveals a Source of Perivascular-Derived Mesenchymal Stem Cells in Zebrafish.
- Published in:
- Stem Cells, 2014, v. 32, n. 10, p. 2767, doi. 10.1002/stem.1758
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- Publication type:
- Article
Concise Review: Transplantation of Human Hematopoietic Cells for Extracellular Matrix Protein Deficiency in Epidermolysis Bullosa.
- Published in:
- Stem Cells, 2011, v. 29, n. 6, p. 900, doi. 10.1002/stem.647
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- Publication type:
- Article
Concise Review: Hitting the Right Spot with Mesenchymal Stromal Cells.
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- Stem Cells, 2010, v. 28, n. 8, p. 1446, doi. 10.1002/stem.459
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- Publication type:
- Article
Sarcoma Derived from Cultured Mesenchymal Stem Cells.
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- Stem Cells, 2007, v. 25, n. 2, p. 371, doi. 10.1634/stemcells.2005-0620
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- Publication type:
- Article
On Medawar's 'Actively acquired tolerance of foreign cells'.
- Published in:
- Experimental Dermatology, 2014, v. 23, n. 2, p. 97, doi. 10.1111/exd.12227
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- Publication type:
- Article
Management of severe epidermolysis bullosa by haematopoietic transplant: principles, perspectives and pitfalls.
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- Experimental Dermatology, 2012, v. 21, n. 12, p. 896, doi. 10.1111/exd.12014
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- Publication type:
- Article
Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy.
- Published in:
- Scientific Reports, 2016, p. 24579, doi. 10.1038/srep24579
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- Publication type:
- Article
Lysyl Hydroxylase 3 Localizes to Epidermal Basement Membrane and Is Reduced in Patients with Recessive Dystrophic Epidermolysis Bullosa.
- Published in:
- PLoS ONE, 2015, v. 10, n. 9, p. 1, doi. 10.1371/journal.pone.0137639
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- Publication type:
- Article
MSC Therapy Attenuates Obliterative Bronchiolitis after Murine Bone Marrow Transplant.
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- PLoS ONE, 2014, v. 9, n. 10, p. 1, doi. 10.1371/journal.pone.0109034
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- Publication type:
- Article
Bone Marrow Stromal and Vascular Smooth Muscle Cells Have Chemosensory Capacity via Bitter Taste Receptor Expression.
- Published in:
- PLoS ONE, 2013, v. 8, n. 3, p. 1, doi. 10.1371/journal.pone.0058945
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- Publication type:
- Article
Cerebrospinal Fluid Matrix Metalloproteinases Are Elevated in Cerebral Adrenoleukodystrophy and Correlate with MRI Severity and Neurologic Dysfunction.
- Published in:
- PLoS ONE, 2012, v. 7, n. 11, p. 1, doi. 10.1371/journal.pone.0050430
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- Publication type:
- Article
Elevated Cerebral Spinal Fluid Cytokine Levels in Boys with Cerebral Adrenoleukodystrophy Correlates with MRI Severity.
- Published in:
- PLoS ONE, 2012, v. 7, n. 2, p. 1, doi. 10.1371/journal.pone.0032218
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- Publication type:
- Article
A 3D in vitro model of the dermoepidermal junction amenable to mechanical testing.
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- Journal of Biomedical Materials Research, Part A, 2018, v. 106, n. 12, p. 3231, doi. 10.1002/jbm.a.36519
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- Publication type:
- Article
Mesenchymal stromal cells inhibit murine syngeneic anti-tumor immune responses by attenuating inflammation and reorganizing the tumor microenvironment.
- Published in:
- Cancer Immunology, Immunotherapy, 2015, v. 64, n. 11, p. 1449, doi. 10.1007/s00262-015-1749-6
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- Publication type:
- Article
372 HMGB1 Localization as a Driver of Carcinogenesis in RDEB-Associated Squamous Cell Carcinoma.
- Published in:
- 2023
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- Publication type:
- Abstract
Pericardial effusion after pediatric hematopoietic cell transplant.
- Published in:
- Pediatric Transplantation, 2013, v. 17, n. 3, p. 294, doi. 10.1111/petr.12062
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- Publication type:
- Article
Rapid Induction of Cerebral Organoids From Human Induced Pluripotent Stem Cells Using a Chemically Defined Hydrogel and Defined Cell Culture Medium.
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- Stem Cells Translational Medicine, 2016, v. 5, n. 7, p. 970, doi. 10.5966/sctm.2015-0305
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- Publication type:
- Article
ABCB5+ dermal mesenchymal stromal cells with favorable skin homing and local immunomodulation for recessive dystrophic epidermolysis bullosa treatment.
- Published in:
- Stem Cells, 2021, v. 39, n. 7, p. 897, doi. 10.1002/stem.3356
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- Publication type:
- Article
Chitotriosidase as a biomarker of cerebral adrenoleukodystrophy.
- Published in:
- 2011
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- Publication type:
- journal article
Comparative evaluation of set-level techniques in predictive classification of gene expression samples.
- Published in:
- BMC Bioinformatics, 2012, v. 13, n. Suppl 10, p. 1, doi. 10.1186/1471-2105-13-S10-S15
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- Publication type:
- Article
Prediction of DNA-binding propensity of proteins by the ball-histogram method using automatic template search.
- Published in:
- BMC Bioinformatics, 2012, v. 13, n. Suppl 10, p. 1, doi. 10.1186/1471-2105-13-S10-S3
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- Publication type:
- Article
From Mesoderm to Mesodermatology: Bone Marrow Mesenchymal Cells Heal Skin Wounds.
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- Molecular Therapy, 2015, v. 23, n. 8, p. 1283, doi. 10.1038/mt.2015.84
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- Publication type:
- Article
From Marrow to Matrix: Novel Gene and Cell Therapies for Epidermolysis Bullosa.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 6, p. 987, doi. 10.1038/mt.2015.47
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- Publication type:
- Article
TALEN-based Gene Correction for Epidermolysis Bullosa.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 6, p. 1151, doi. 10.1038/mt.2013.56
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- Publication type:
- Article
Genes and compounds that increase type VII collagen expression as potential treatments for dystrophic epidermolysis bullosa.
- Published in:
- Experimental Dermatology, 2022, v. 31, n. 7, p. 1065, doi. 10.1111/exd.14555
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- Publication type:
- Article
Future applications of 3D bioprinting: A promising technology for treating recessive dystrophic epidermolysis bullosa.
- Published in:
- Experimental Dermatology, 2022, v. 31, n. 3, p. 384, doi. 10.1111/exd.14484
- By:
- Publication type:
- Article
Role of transforming growth factor‐β1 in recessive dystrophic epidermolysis bullosa squamous cell carcinoma.
- Published in:
- Experimental Dermatology, 2021, v. 30, n. 5, p. 664, doi. 10.1111/exd.14304
- By:
- Publication type:
- Article
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes.
- Published in:
- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-50595-1
- By:
- Publication type:
- Article
Healthcare access, satisfaction, and health-related quality of life among children and adults with rare diseases.
- Published in:
- 2022
- By:
- Publication type:
- journal article
Abcg2‐expressing side population cells contribute to cardiomyocyte renewal through fusion.
- Published in:
- FASEB Journal, 2020, v. 34, n. 4, p. 5642, doi. 10.1096/fj.201902105R
- By:
- Publication type:
- Article
Analysis of Glycosaminoglycans in Cerebrospinal Fluid from Patients with Mucopolysaccharidoses by Isotope-Dilution Ultra-Performance Liquid Chromatography--Tandem Mass Spectrometry.
- Published in:
- Clinical Chemistry, 2011, v. 57, n. 7, p. 1005, doi. 10.1373/clinchem.2010.161141
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- Publication type:
- Article
CRISPR/Cas9-Based Lateral Flow and Fluorescence Diagnostics.
- Published in:
- Bioengineering (Basel), 2021, v. 8, n. 2, p. 1, doi. 10.3390/bioengineering8020023
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- Publication type:
- Article
Lipid Composition of Whole Brain and Cerebellum in Hurler Syndrome (MPS IH) Mice.
- Published in:
- Neurochemical Research, 2011, v. 36, n. 9, p. 1669, doi. 10.1007/s11064-011-0400-y
- By:
- Publication type:
- Article
Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa.
- Published in:
- Journal of Investigative Dermatology, 2015, v. 135, n. 9, p. 2319, doi. 10.1038/jid.2015.158
- By:
- Publication type:
- Article
Peripheral Neuro-Immune Pathology in Recessive Dystrophic Epidermolysis Bullosa.
- Published in:
- Journal of Investigative Dermatology, 2015, v. 135, n. 4, p. 1193, doi. 10.1038/jid.2014.500
- By:
- Publication type:
- Article
Augmentation of Cutaneous Wound Healing by Pharmacologic Mobilization of Endogenous Bone Marrow Stem Cells.
- Published in:
- Journal of Investigative Dermatology, 2014, v. 134, n. 9, p. 2312, doi. 10.1038/jid.2014.209
- By:
- Publication type:
- Article