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Hepatic stem cells: from inside and outside the liver?

Cell proliferation, 2004-02, Vol.37 (1), p.1-21

2004

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The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

Journal of inherited metabolic disease, 2016-07, Vol.39 (4), p.499-512

2016

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A nonmyeloablative chimeric mouse model accurately defines microglia and macrophage contribution in glioma

Neuropathology and applied neurobiology, 2019-02, Vol.45 (2), p.119-140

2019

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Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

Gene therapy, 2006-01, Vol.13 (2), p.117-126

2006

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Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Haematologica (Roma), 2012-09, Vol.97 (9), p.1320-1328

2012

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Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy

Gene therapy, 2004-07, Vol.11 (14), p.1117-1125

2004

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Is it congenital or acquired von Willebrands disease?

Haemophilia : the official journal of the World Federation of Hemophilia, 2015-01, Vol.21 (1), p.e113-e115

2015

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In vivo T-cell depletion using alemtuzumab in family and unrelated donor transplantation for pediatric non-malignant disease achieves engraftment with low incidence of graft vs. host disease

Pediatric transplantation, 2015-03, Vol.19 (2), p.211-218

2015

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Hyperactive behaviour in the mouse model of mucopolysaccharidosis IIIB in the open field and home cage environments

Genes, brain and behavior, 2011-08, Vol.10 (6), p.673-682

2011

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An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy

The Journal of biological chemistry, 2001-06, Vol.276 (25), p.23018-23027

2001

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Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model

Brain (London, England : 1878), 2015-02, Vol.138 (Pt 2), p.336-355

2015

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Bile acid biosynthesis in Smith-Lemli-Opitz syndrome bypassing cholesterol: Potential importance of pathway intermediates

The Journal of steroid biochemistry and molecular biology, 2021-02, Vol.206, p.105794-105794, Article 105794

2021

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Sterols and oxysterols in plasma from Smith-Lemli-Opitz syndrome patients

The Journal of steroid biochemistry and molecular biology, 2017-05, Vol.169, p.77-87

2017

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Hematopoietic stem cell transplant for the mucopolysaccharidoses

Expert opinion on orphan drugs, 2016-04, Vol.4 (4), p.379-393

2016

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Lentiviral Expression of SGSH Under the Human CD11b Promoter in transplanted Thaematopoietic Stem Cells Fully Corrects Behaviour and Neuropathology of the Mouse Model of Mucopolysaccharidosis IIIA

Molecular therapy, 2012-09, Vol.20 (9), p.17-17

2012

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Comparison of HIV- and EIAV-Based Vectors on Their Efficiency in Transducing Murine and Human Hematopoietic Repopulating Cells

Molecular therapy, 2005-09, Vol.12 (3), p.537-546

2005

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THE PREVENTION OF EPISOTIC TREMORS IN ANIMALS WITH A PORTACAVAL SHUNT

The American surgeon, 1964-12, Vol.30, p.856

1964

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Is it congenital or acquired von Willebrands disease?

Haemophilia : the official journal of the World Federation of Hemophilia, 2015, Vol.21 (1), p.e113-e115

2015

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Genetic Modification of Haematopoietic Stem Cells. Methods and Protocols

British Journal of Biomedical Science, 2009, Vol.66 (4), p.223

2009

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Book Review

British Journal of Biomedical Science, 2009, Vol.66 (4), p.223-224

2009

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Book Review

British Journal of Biomedical Science, 2009, Vol.66 (4), p.223-224

2009

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A Review of Property Enhancement Techniques for Carrageenan-based Films and Coatings

Carbohydrate polymers, 2019-07, Vol.216, p.287-302

2019

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Long-Term Effects of Intensive Glucose Lowering on Cardiovascular Outcomes

The New England journal of medicine, 2011-03, Vol.364 (9), p.818-828

2011

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Effects of Combination Lipid Therapy in Type 2 Diabetes Mellitus

The New England journal of medicine, 2010-04, Vol.362 (17), p.1563-1574

2010

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Hepatic stem cells: from inside and outside the liver?

The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

A nonmyeloablative chimeric mouse model accurately defines microglia and macrophage contribution in glioma

Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy

Is it congenital or acquired von Willebrands disease?

In vivo T-cell depletion using alemtuzumab in family and unrelated donor transplantation for pediatric non-malignant disease achieves engraftment with low incidence of graft vs. host disease

Hyperactive behaviour in the mouse model of mucopolysaccharidosis IIIB in the open field and home cage environments

An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy

Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model

Bile acid biosynthesis in Smith-Lemli-Opitz syndrome bypassing cholesterol: Potential importance of pathway intermediates

Sterols and oxysterols in plasma from Smith-Lemli-Opitz syndrome patients

Hematopoietic stem cell transplant for the mucopolysaccharidoses

Lentiviral Expression of SGSH Under the Human CD11b Promoter in transplanted Thaematopoietic Stem Cells Fully Corrects Behaviour and Neuropathology of the Mouse Model of Mucopolysaccharidosis IIIA

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy

Comparison of HIV- and EIAV-Based Vectors on Their Efficiency in Transducing Murine and Human Hematopoietic Repopulating Cells

THE PREVENTION OF EPISOTIC TREMORS IN ANIMALS WITH A PORTACAVAL SHUNT

Is it congenital or acquired von Willebrands disease?

Genetic Modification of Haematopoietic Stem Cells. Methods and Protocols

Book Review

Book Review

A Review of Property Enhancement Techniques for Carrageenan-based Films and Coatings

Long-Term Effects of Intensive Glucose Lowering on Cardiovascular Outcomes

Effects of Combination Lipid Therapy in Type 2 Diabetes Mellitus

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