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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

PloS one, 2010-12, Vol.5 (12), p.e14192-e14192

2010

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Study of Telomere Length Reveals Rapid Aging of Human Marrow Stromal Cells following In Vitro Expansion

Stem cells (Dayton, Ohio), 2004-09, Vol.22 (5), p.675-682

2004

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Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

PloS one, 2012-04, Vol.7 (4), p.e35787-e35787

2012

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A small proportion of mesenchymal stem cells strongly expresses functionally active CXCR4 receptor capable of promoting migration to bone marrow

Blood, 2004-11, Vol.104 (9), p.2643-2645

2004

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Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

The Journal of pediatrics, 2009-04, Vol.154 (4), p.609-611

2009

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Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB

Behavioural brain research, 2010-06, Vol.209 (2), p.212-220

2010

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Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

PloS one, 2013-10, Vol.8 (10), p.e77632

2013

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Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Acta pædiatrica (Oslo), 2005-07, Vol.94 (7), p.872-877

2005

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Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

Journal of inherited metabolic disease, 2011-04, Vol.34 (2), p.499-508

2011

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High dose genistein in Sanfilippo syndrome: A randomised controlled trial

Journal of inherited metabolic disease, 2021-09, Vol.44 (5), p.1248-1262

2021

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Thoracolumbar Kyphosis in Treated Mucopolysaccharidosis 1 (Hurler Syndrome)

Spine (Philadelphia, Pa. 1976), 2014-03, Vol.39 (5), p.381-387

2014

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Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

Molecular genetics and metabolism, 2009-11, Vol.98 (3), p.235-242

2009

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Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

Molecular genetics and metabolism, 2010-03, Vol.99 (3), p.269-274

2010

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Retrovirally mediated correction of bone marrow–derived mesenchymal stem cells from patients with mucopolysaccharidosis type I

Blood, 2002-03, Vol.99 (5), p.1857-1859

2002

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Enzyme Replacement Therapy and Extended Newborn Screening for Mucopolysaccharidoses: Opinions of Treating Physicians

JIMD Reports - Case and Research Reports, 2011/1, 2011-01, Vol.1, p.9-15

2011

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Limitations of enzyme replacement therapy: Current and future

Journal of inherited metabolic disease, 2006-04, Vol.29 (2-3), p.442-447

Wraith, J. E.

2006

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The majority of MPS I patients tested raise inhibitory allo-antibodies against enzyme replacement therapy

Molecular genetics and metabolism, 2013-02, Vol.108 (2), p.S81-S81

2013

Volltextzugriff (PDF)

Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I

Blood, 2011-11, Vol.118 (21), p.4010-4010

2011

Volltextzugriff (PDF)

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Acta Paediatrica, 2005-07, Vol.94 (7), p.872-877

2005

Volltextzugriff (PDF)

Variant Gaucher disease characterized by dysmorphic features, absence of cardiovascular involvement, laryngospasm, and compound heterozygosity for a novel mutation (D409H/C16S)

American journal of medical genetics, 2002-05, Vol.109 (4), p.328-331

2002

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Frequent deletions at Xq28 indicate genetic heterogeneity in Hunter syndrome

Human genetics, 1991-03, Vol.86 (5), p.505-508

1991

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Signal One and Two Blockade Are Both Critical for Non-Myeloablative Murine HSCT across a Major Histocompatibility Complex Barrier: e77632

PloS one, 2013-10, Vol.8 (10)

2013

Volltextzugriff (PDF)

Novel Mutations in Sanfilippo a Syndrome: Implications for Enzyme function

Human molecular genetics, 1997-09, Vol.6 (9), p.1573-1579

1997

Volltextzugriff (PDF)

Mucopolysaccharidoses and Oligosaccharidoses

Inborn Metabolic Diseases, p.495-507

Volltextzugriff (PDF)

Treatment: Present Status and New Trends

Inborn Metabolic Diseases, p.81-97

Volltextzugriff (PDF)

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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease

Study of Telomere Length Reveals Rapid Aging of Human Marrow Stromal Cells following In Vitro Expansion

Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

A small proportion of mesenchymal stem cells strongly expresses functionally active CXCR4 receptor capable of promoting migration to bone marrow

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB

Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

High dose genistein in Sanfilippo syndrome: A randomised controlled trial

Thoracolumbar Kyphosis in Treated Mucopolysaccharidosis 1 (Hurler Syndrome)

Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

Retrovirally mediated correction of bone marrow–derived mesenchymal stem cells from patients with mucopolysaccharidosis type I

Enzyme Replacement Therapy and Extended Newborn Screening for Mucopolysaccharidoses: Opinions of Treating Physicians

Limitations of enzyme replacement therapy: Current and future

The majority of MPS I patients tested raise inhibitory allo-antibodies against enzyme replacement therapy

Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I

Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I

Variant Gaucher disease characterized by dysmorphic features, absence of cardiovascular involvement, laryngospasm, and compound heterozygosity for a novel mutation (D409H/C16S)

Frequent deletions at Xq28 indicate genetic heterogeneity in Hunter syndrome

Signal One and Two Blockade Are Both Critical for Non-Myeloablative Murine HSCT across a Major Histocompatibility Complex Barrier: e77632

Novel Mutations in Sanfilippo a Syndrome: Implications for Enzyme function

Mucopolysaccharidoses and Oligosaccharidoses

Treatment: Present Status and New Trends

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