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Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

Immune Mediated Cytopenia's Following Cord Blood Transplantation for Hurler's Syndrome: A Failure of Pre-Transplant Immune Suppression

David Deambrosis, MBBS, FRACP, Ryan Hum, Prashant Hiwarkar, Kay Poulton, Denise Bonney, Helen Jane Campbell, FRCP, MD FRCPath, Robert Wynn, MD

Blood (2018) 132 (Supplement 1): 3374.

https://doi.org/10.1182/blood-2018-99-112995

Published: 2018

Journal Articles

The Changing Patterns of Graft Failure in MPS1H, Hurler Syndrome: A Review of 30-Years Experience

Su Han Lum, Weston P Miller, Simon Jones, Jean Mercer, Troy Lund, Denise Bonney, Paul J Orchard, Robert Wynn

Blood (2016) 128 (22): 4700.

https://doi.org/10.1182/blood.V128.22.4700.4700

Published: 2016

Journal Articles

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

Melisa A. Vance, PhD, Matthew Hirsch, PhD, Richard Jude Samulski, PhD, Joanne Kurtzberg, MD, Laurie Goodrich, DVM, PhD ACVS Diplomate, Llanga Telmo, Bsc

Blood (2015) 126 (23): 2041.

https://doi.org/10.1182/blood.V126.23.2041.2041

Published: 2015

Journal Articles

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

Blood (2015) 125 (13): 2164–2172.

https://doi.org/10.1182/blood-2014-11-608075

Published: 2015

Includes: Supplemental data

Journal Articles

Long-term outcomes in MPS-IH: throwing stars

Michael A. Pulsipher

Blood (2015) 125 (13): 2016–2017.

https://doi.org/10.1182/blood-2015-02-626507

Published: 2015

Journal Articles

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

Alice Pievani, PhD, Isabella Maria Rebecca Azario, Laura Antolini, Tsutomu Shimada, Pravin Patel, Cristina Remoli, Benedetta Rambaldi, Maria Grazia Valsecchi, Mara Riminucci, Andrea Biondi, Shunji Tomatsu, Marta Serafini

Blood (2014) 124 (21): 649.

https://doi.org/10.1182/blood.V124.21.649.649

Published: 2014

Journal Articles

Better BMT for Hurler syndrome—on the level?

Michael A. Pulsipher

Blood (2013) 121 (19): 3785–3787.

https://doi.org/10.1182/blood-2013-04-491779

Published: 2013

Journal Articles

Platelets: An Efficient Depot for Generation and Distribution of Lysosomal Idua in Enzyme-Deficient MPS I Mice.

Mei Dai, PhD, Phuong Cao, Dao Pan, PhD

Blood (2012) 120 (21): 3157.

https://doi.org/10.1182/blood.V120.21.3157.3157

Published: 2012

Journal Articles

Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

Mieke Aldenhoven, Maria Escolar, Robert Wynn, MRCP, FRCPath, MD, Ed Wraith, Anne O'Meara, Paul Veys, Nizar Mahlaoui, MD, Michele Poe, Paul Orchard, MD, Joanne Kurtzberg, MD, Jaap J Boelens, MD, PhD

Blood (2012) 120 (21): 1958.

https://doi.org/10.1182/blood.V120.21.1958.1958

Published: 2012

Journal Articles

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

H. Angharad Watson, PhD, Rebecca J Holley, PhD, Kia J Langford-Smith, Fiona L Wilkinson, PhD, Toin H van Kuppevelt, Robert Wynn, MD, J. Ed Wraith, Catherine L R Merry, Brian W Bigger, PhD

Blood (2011) 118 (21): 4010.

https://doi.org/10.1182/blood.V118.21.4010.4010

Published: 2011

Journal Articles

High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice.

Daren Wang, PhD, Wei Zhang, MS, Theodosia A. Kalfa, MD, PhD, Punam Malik, MD, Dao Pan, PhD

Blood (2008) 112 (11): 2355.

https://doi.org/10.1182/blood.V112.11.2355.2355

Published: 2008

Journal Articles

Patient-Specific Induced Pluripotent Stem Cells in Hurler Syndrome

Jakub Tolar, MD, PhD, In-Hyun Park, M.D., Lily Xia, B.S., Mark Osborn, B.S., Ron T. McElmurry, B.S., Paul J. Orchard, M.D., George Q. Daley, MD, PhD, Bruce R Blazar, M.D.

Blood (2008) 112 (11): 386.

https://doi.org/10.1182/blood.V112.11.386.386

Published: 2008

Journal Articles

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Jakub Tolar, MD, PhD, Paul J. Orchard, M.D., Nigel Key, M.D., Bruce R. Blazar, M.D.

Blood (2007) 110 (11): 3951.

https://doi.org/10.1182/blood.V110.11.3951.3951

Published: 2007

Journal Articles

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Jakub Tolar, MD, PhD, Paul J. Orchard, M.D., Nigel Key, M.D., Bruce R. Blazar, M.D.

Blood (2007) 110 (11): 2160.5.

https://doi.org/10.1182/blood.V110.11.2160.5.2160.5

Published: 2007

Journal Articles

Heparin and Endogenous Heparan Sulfate Modulate Bone Morphogenetic Protein-4 (BMP-4) Signaling and Activity.

Shaukat A. Khan, PhD, Matthew S. Nelson, BS, Chendong Pan, MS, MD, Patrick M. Gaffney, MD, Pankaj Gupta, MD

Blood (2006) 108 (11): 4230.

https://doi.org/10.1182/blood.V108.11.4230.4230

Published: 2006

Journal Articles

Monitoring Graft Function after First Allogeneic Transplant for Mucopolysaccharidosis-IH: Patterns of Rejection and Outcome of Second Allograft Procedure.

Sonya Zaman, Amy Sutton, Heather Church, Wraith Ed, Carr Trevor, Grainger John, Wynn Robert

Blood (2005) 106 (11): 2058.

https://doi.org/10.1182/blood.V106.11.2058.2058

Published: 2005

Journal Articles

Neonatal Gene Transfer Induces Tolerance to Human Factor VIII in Mice and Cats.

Lingfei Xu, MD, Manxue Mei, MS, Mark E. Haskins, VMD, PhD, Karyn Cullen, Katherine P. Ponder, MD

Blood (2005) 106 (11): 215.

https://doi.org/10.1182/blood.V106.11.215.215

Published: 2005

Journal Articles

Safety and Efficacy of Enzyme Replacement Therapy (ERT) in Combination with Haematopoietic Stem Cell Transplantation (HSCT) for Hurler’s Syndrome: European Experience 2003–2005.

Jaap Jan Boelens, MD, PhD, R. Wynn, MD, E. Wraith, MD, A. O’Mearra, MD, P. Veys, MD, N. Wulffraat, MD, PhD

Blood (2005) 106 (11): 5307.

https://doi.org/10.1182/blood.V106.11.5307.5307

Published: 2005

Journal Articles

Characterization of an Immunodeficient Mucopolysaccharidosis Type I (MPS-I) Mouse Model Suitable for Preclinical Testing of Human Stem Cell and Gene Therapy.

Mayra F. Quito-Rivera, BS, Leah E. Colvin-Wanshura, BS, Matthew S. Nelson, BS, Zhenhong Nan, PhD, Shaukat A. Khan, PhD, Tyson B. Rogers, MS, Indrani Maitra, PhD, Walter C. Low, PhD, Pankaj Gupta, MD

Blood (2005) 106 (11): 1265.

https://doi.org/10.1182/blood.V106.11.1265.1265

Published: 2005

NARROW

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Immune Mediated Cytopenia's Following Cord Blood Transplantation for Hurler's Syndrome: A Failure of Pre-Transplant Immune Suppression

The Changing Patterns of Graft Failure in MPS1H, Hurler Syndrome: A Review of 30-Years Experience

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

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

Long-term outcomes in MPS-IH: throwing stars

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

Better BMT for Hurler syndrome—on the level?

Platelets: An Efficient Depot for Generation and Distribution of Lysosomal Idua in Enzyme-Deficient MPS I Mice.

Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

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

High Level Production of a Lysosomal Enzyme from HSC-Derived Erythroid Cells for Therapeutic Correction of Hurler Syndrome in Mice.

Patient-Specific Induced Pluripotent Stem Cells in Hurler Syndrome

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Heparin and Endogenous Heparan Sulfate Modulate Bone Morphogenetic Protein-4 (BMP-4) Signaling and Activity.

Monitoring Graft Function after First Allogeneic Transplant for Mucopolysaccharidosis-IH: Patterns of Rejection and Outcome of Second Allograft Procedure.

Neonatal Gene Transfer Induces Tolerance to Human Factor VIII in Mice and Cats.

Safety and Efficacy of Enzyme Replacement Therapy (ERT) in Combination with Haematopoietic Stem Cell Transplantation (HSCT) for Hurler’s Syndrome: European Experience 2003–2005.

Characterization of an Immunodeficient Mucopolysaccharidosis Type I (MPS-I) Mouse Model Suitable for Preclinical Testing of Human Stem Cell and Gene Therapy.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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