High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector

Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N -sulfoglucosamine sulfohydrolase (SGSH) protein, a sulfamidase required for the degradation of heparan sulfate glycosaminoglycans (GAGs). The accumulat...

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Veröffentlicht in:Gene therapy 2014-12, Vol.21 (12), p.1001-1007
Hauptverfasser: Quiviger, M, Arfi, A, Mansard, D, Delacotte, L, Pastor, M, Scherman, D, Marie, C
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container_issue 12
container_start_page 1001
container_title Gene therapy
container_volume 21
creator Quiviger, M
Arfi, A
Mansard, D
Delacotte, L
Pastor, M
Scherman, D
Marie, C
description Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N -sulfoglucosamine sulfohydrolase (SGSH) protein, a sulfamidase required for the degradation of heparan sulfate glycosaminoglycans (GAGs). The accumulation of these macromolecules leads to somatic organ pathologies, severe neurodegeneration and death. To assess a novel gene therapy approach based on prolonged secretion of the missing enzyme by the liver, mediated by hydrodynamic gene delivery, we first compared a kanamycin and an antibiotic-free expression plasmid vector, called pFAR4. Thanks to the reduced vector size, pFAR4 derivatives containing either a ubiquitous or a liver-specific promoter mediated a higher reporter gene expression level than the control plasmid. Hydrodynamic delivery of SGSH-encoding pFAR4 into MPS-IIIA diseased mice led to high serum levels of sulfamidase protein that was efficiently taken up by neighboring organs, as shown by the correction of GAG accumulation. A similar reduction in GAG content was also observed in the brain, at early stages of the disease. Thus, this study contributes to the effort towards the development of novel biosafe non-viral gene vectors for therapeutic protein expression in the liver, and represents a first step towards an alternative gene therapy approach for the MPS-IIIA disease.
doi_str_mv 10.1038/gt.2014.75
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subjects 692/699/317
692/700/565/201
Animals
Antibiotics
Biomedical and Life Sciences
Biomedicine
Biotechnology
Care and treatment
Cell Biology
Disease Models, Animal
DNA Copy Number Variations
Expression vectors
Gene Expression
Gene Therapy
Gene transfer
Gene Transfer Techniques
Genetic aspects
Genetic disorders
Genetic research
Genetic Therapy - methods
Genetic Vectors
Glycosaminoglycans
Glycosaminoglycans - metabolism
Heparan sulfate
Human Genetics
Hydrolases - genetics
Hydrolases - secretion
Kanamycin
Life Sciences
Liver
Liver - enzymology
Macromolecules
Mice
Mucopolysaccharidosis
Mucopolysaccharidosis III - genetics
Mucopolysaccharidosis III - therapy
Nanotechnology
Neurodegeneration
original-article
Plasmids - genetics
Properties
Protein biosynthesis
Protein expression
Proteins
Reporter gene
Rodents
Secretion
Serum levels
Vectors (Biology)
title High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector
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