(Q40210766)

18 September 2017

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy (English)

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18 September 2017

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mucopolysaccharidosis I

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7

Meg M Sleeper

1 reference

18 September 2017

Anne M Traas

1

1 reference

18 September 2017

Ping Wang

2

1 reference

18 September 2017

Xiucui Ma

3

1 reference

18 September 2017

Mindy Tittiger

4

1 reference

18 September 2017

Laura Schaller

5

1 reference

18 September 2017

Patricia O'donnell

6

1 reference

18 September 2017

Charles Vite

8

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18 September 2017

Ramin Herati

9

1 reference

18 September 2017

Gustavo D Aguirre

10

1 reference

18 September 2017

Mark Haskins

11

1 reference

18 September 2017

Katherine P Ponder

12

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18 September 2017

22 May 2007

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18 September 2017

1 reference

18 September 2017

15

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18 September 2017

8

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18 September 2017

1423-1431

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Architecture of the canine IDUA gene and mutation underlying canine mucopolysaccharidosis I.

18 September 2017

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Follow-up of nine patients with Hurler syndrome after bone marrow transplantation.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Usefulness of bone marrow transplantation in the Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Mucopolysaccharidoses and the eye.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Identification and characterization of 13 new mutations in mucopolysaccharidosis type I patients.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Enzyme replacement in a canine model of Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

The heart in the Hurler syndrome: gross, histologic and ultrastructural observations in five necropsy cases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Cardiac manifestations in the mouse model of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Cardiac functional and histopathologic findings in humans and mice with mucopolysaccharidosis type I: implications for assessment of therapeutic interventions in hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Gene therapy of the brain in the dog model of Hurler's syndrome.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Newborn screening for lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Tandem mass spectrometric analysis of dried blood spots for screening of mucopolysaccharidosis I in newborns

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300201

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300201

1 reference

18 September 2017

1 reference