Lentiviral-Mediated Gene Therapy Results in Sustained Expression of β-Glucuronidase for up to 12 Months in the Gusmps/mps and up to 18 Months in the Gustm(L175F)Sly Mouse Models of Mucopolysaccharidosis Type VII-Reference-Cited by-同舟云学术

Lentiviral-Mediated Gene Therapy Results in Sustained Expression of β-Glucuronidase for up to 12 Months in the Gusmps/mps and up to 18 Months in the Gustm(L175F)Sly Mouse Models of Mucopolysaccharidosis Type VII

Published:2014-09 Issue:9 Volume:25 Page:798-810
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Derrick-Roberts Ainslie L.K.¹²,Pyragius Carmen E.¹,Kaidonis Xenia M.¹³,Jackson Matilda R.¹³,Anson Donald S.¹²,Byers Sharon¹²³

Affiliation:

1. Genetics and Molecular Pathology, South Australia Pathology (Children, Youth and Women's Health Service Site), Adelaide, South Australia 5006, Australia.

2. Discipline of Paediatrics, The University of Adelaide, Adelaide, South Australia 5000, Australia.

3. Department of Genetics, School of Molecular and Biomedical Science, The University of Adelaide, Adelaide, South Australia 5000, Australia.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.141

Reference43 articles.

1. Phenotypic expression in mucopolysaccharidosis VII.

2. Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII

3. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency.

4. Reversal of Pathology in the Entire Brain of Mucopolysaccharidosis Type VII Mice after Lentivirus-Mediated Gene Transfer

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