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, In order to identify the optimal vector for gene delivery to the liver, the murine secreted embryonic alkaline phosphatase (mSeAP) gene (CpG free, Invivogen, Toulouse, France) was first introduced into either the pFAR4 pVAX vectors (Invitrogen, Illkirch, France) with the MCS (multiple cloning sites) of pFAR4. 8 Following identification of the optimal eukaryotic expression vector, the murine SeAP gene on pFAR4-hAAT-mSeAP was substituted by the murine sulfamidase (SGSH) cDNA, to give pFAR4-hAAT-SGSH and subsequently pKAR4-hAAT-SGSH
, The 20 ?l reaction mixtures contained 1 ? 10 ng of DNA and 500 mM of each primer. The PCR program used was 1 min at 95°C (5 s at 95°C and 30 s at 60°C) 40x . Standard amplification curves were generated, in the presence of 1-10 ng of control genomic DNA, with a serial dilution of templates, The primer sequences were: pSGSH-F: 5?-AGTGAATGATCCCCCTGATCT and pSGSH-R: 5?-CTCCGT CATCCGCAACTATCA; pSEAP-F: 5?-AGTGAATGATCCCCCTGATCT and pSEAP-R: 5?-AAGCCCAGCAATAGCAGACA for plasmid quantification that was normalized with mouse GAPDH sequence amplified with the following primers: gGAPDH-F3: 5?-CCTGGGATTAGGGTTGGAAAC and gGAPDH-R3: 5?-GCTCAAAGGGCAAGGCTAAAG
, Quantification of enzymatic activities and glycosaminoglycan contents Sera were collected by retro-orbital or intra-cardiac punctures
, The Netherlands), essentially as described in Karpova et al., 44 either directly from serum or after organ grounding and cell lysis. The SGSH activity was finally normalized against the total amount of protein quantified using bicinchoninic acid (BCA) (Pierce BCA Protein Assay Kit, Interleukin-2 and tumor necrosis factor-? levels were quantified using the Quantikine ELISA mouse IL-12 p70 and TNF-? Immunoassay kits, respectively (R&D Systems
, Histological analyses Ten ?m sections of brains frozen in cooled isopentane and taken from agematched wild-type, diseased or treated MPS-IIIA mice (n = 3 per group) were simultaneously stained with either of the following antibodies: rat monoclonal anti-LAMP1 (1D4B, sc-19992, Either biotinylated goat anti-rat antibody (559286, BD Biosciences, Le Pont de Claix, France) or goat anti-rabbit IgG-Peroxidase antibodies (A0545
, Statistical analysis Data were analyzed using GraphPad Prism 5 software. The significance of differences between groups was determined based on exact two-tailed P values obtained with the nonparametric Mann-Whitney test
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