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BCSH/BSBMT guideline: diagnosis and management of veno‐occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation

British journal of haematology, 2013-11, Vol.163 (4), p.444-457

2013

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CTP synthase 1 deficiency in humans reveals its central role in lymphocyte proliferation

Nature (London), 2014-06, Vol.510 (7504), p.288-292

2014

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Hematopoietic stem cell transplantation for CTLA4 deficiency

Journal of allergy and clinical immunology, 2016-08, Vol.138 (2), p.615-619.e1

2016

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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

Frontiers in pediatrics, 2019-10, Vol.7, p.433-433

2019

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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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Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Orphanet journal of rare diseases, 2021-05, Vol.16 (1), p.235-235, Article 235

2021

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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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Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

Orphanet journal of rare diseases, 2011-08, Vol.6 (1), p.55-55, Article 55

2011

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Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

Molecular therapy, 2013-10, Vol.21 (10), p.1938-1949

2013

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Successful cure of C1q deficiency in human subjects treated with hematopoietic stem cell transplantation

Journal of allergy and clinical immunology, 2014-01, Vol.133 (1), p.265-267

2014

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Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice

Molecular therapy, 2012-08, Vol.20 (8), p.1610-1621

2012

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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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Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

The Journal of pediatrics, 2009, Vol.154 (1), p.135-139

2009

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Current International Perspectives on Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders

The Pediatric clinics of North America, 2010-02, Vol.57 (1), p.123-145

2010

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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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Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis

Blood advances, 2020-05, Vol.4 (9), p.1998-2010

2020

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Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test

PloS one, 2011-10, Vol.6 (10), p.e25717-e25717

2011

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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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Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Blood, 2015-03, Vol.125 (13), p.2164-2172

2015

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Haematopoietic stem cell transplantation in inborn errors of metabolism

Current opinion in hematology, 2016-11, Vol.23 (6), p.530-535

2016

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Transplantation in inborn errors of metabolism: current considerations and future perspectives

British journal of haematology, 2014-11, Vol.167 (3), p.293-303

2014

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Burden of adenoviraemia predicts survival in paediatric recipients of allogeneic haematopoietic stem cell transplant

Journal of clinical virology, 2020-06, Vol.127, p.104373-104373, Article 104373

2020

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Wiskott Aldrich Syndrome-2 Caused by Novel Wiskott Aldrich Syndrome Protein–Interacting Protein (WIP) Deficiency Is Associated with Juvenile Myelomonocytic Leukaemia — a Case Report

Journal of clinical immunology, 2023, Vol.43 (1), p.82-84

2023

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Successful Management of Hepatosplenic Infection Due to Saccharomyces cerevisiae in a Child With Acute Lymphoblastic Leukemia

Journal of pediatric hematology/oncology, 2020-03, Vol.42 (2), p.e117

2020

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BCSH/BSBMT guideline: diagnosis and management of veno‐occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation

CTP synthase 1 deficiency in humans reveals its central role in lymphocyte proliferation

Hematopoietic stem cell transplantation for CTLA4 deficiency

Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

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

Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

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

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD)

Successful cure of C1q deficiency in human subjects treated with hematopoietic stem cell transplantation

Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice

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

Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

Current International Perspectives on Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders

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

Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis

Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test

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

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Haematopoietic stem cell transplantation in inborn errors of metabolism

Transplantation in inborn errors of metabolism: current considerations and future perspectives

Burden of adenoviraemia predicts survival in paediatric recipients of allogeneic haematopoietic stem cell transplant

Wiskott Aldrich Syndrome-2 Caused by Novel Wiskott Aldrich Syndrome Protein–Interacting Protein (WIP) Deficiency Is Associated with Juvenile Myelomonocytic Leukaemia — a Case Report

Successful Management of Hepatosplenic Infection Due to Saccharomyces cerevisiae in a Child With Acute Lymphoblastic Leukemia

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