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Research article

First published 2012-7

Intracerebroventricular Transplantation of Human Bone Marrow-Derived Multipotent Progenitor Cells in an Immunodeficient Mouse Model of Mucopolysaccharidosis Type I (MPS-I)

Zhenhong Nan, Laurie Shekels, Oleg Ryabinin [...]

Cell Transplantation

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Case report

First published 2017-03

Successful Desensitization in a Patient with Hypersensitivity Reaction to Laronidase

İnsu Yılmaz, Aslı Sezgin Çağlar, Fatih Kardas [...]

Pediatric Allergy,Immunology,and Pulmonology

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Research article

First published 1992-08

Correction of Mucopolysaccharidosis Type I Fibroblasts by Retroviral-Mediated Transfer of the Human α- l -Iduronidase Gene

D. S. Anson, J. Bielicki, J. J. Hopwood

Human Gene Therapy

Open access

Research article

First published 2015-3

Airway-Related Symptoms and Surgeries in Patients With Mucopolysaccharidosis I

Pamela Arn, Iain A. Bruce, James E. Wraith [...]

Annals of Otology,Rhinology & Laryngology

Free access

Research article

First published 2011-5

Histopathologic Changes of the Ear in Canine Models of Mucopolysaccharidosis Types I and VII

J. Hordeaux, J. Deniaud, I. Bemelmans [...]

Veterinary Pathology

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Research article

First published 2012-06

Hurler Disease Bone Marrow Stromal Cells Exhibit Altered Ability to Support Osteoclast Formation

Francesca Gatto, Daniela Redaelli, Agnese Salvadè [...]

Stem Cells and Development

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Research article

First published 1998-12

Mobilization and Transduction of Peripheral Blood Progenitor Cells in Patients with Mucopolysaccharidosis I

Allison Hubel, David Stroncek, Dao Pan [...]

Stem Cells and Development

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Research article

First published 1999-09

Enzymatic Correction and Cross-Correction of Mucopolysaccharidosis Type I Fibroblasts by Adeno-Associated Virus-Mediated Transduction of the alpha-L-Iduronidase Gene

Seth D. Hartung, Rukmini G. Reddy, Chester B. Whitley [...]

Human Gene Therapy

Open access

Case report

First published 2023

Left-sided valvular heart disease and retinopathy in a 38-year-old woman with attenuated mucopolysaccharidosis: a case report

Faizal Z. Asumda, Jessica A. Kraker, Sarah C. Thomas [...]

Therapeutic Advances in Rare Disease

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Research article

First published 1999-06

Gene Therapy for Canine alpha-L-Iduronidase Deficiency: In Utero Adoptive Transfer of Genetically Corrected Hematopoietic Progenitors Results in Engraftment but Not Amelioration of Disease

Carolyn Lutzko, Fusayuki Omori, Anthony C. G. Abrams-Ogg [...]

Human Gene Therapy

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