Journal: Molecular Genetics and Metabolism Reports

Article Title: Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models

doi: 10.1016/j.ymgmr.2023.101011

Figure Lengend Snippet: Lysotracker visualization of lysosomes in MPS I and MPS VI fibroblasts and effect of odiparcil treatment. A. Representative images of lysotracker labelling in healthy (control) and MPS I fibroblasts. B, C. Quantification of fluorescence of lysotracker in control and MPS I (B) or MPS VI (C) fibroblasts. D, E. Representative images of MPS VI (D) or MPS I (E) fibroblasts treated with vehicle (DMSO) or odiparcil. F, G. Quantification of fluorescence of lysotracker after odiparcil treatment in MPS VI (F) or MPS I (G) fibroblasts, normalized to the DMSO vehicle control. Data is presented as mean ± SEM. Groups were statistically analysed using a one-way ANOVA followed by Dunnett's test; * p < 0.05, ** p < 0.01, *** p < 0.001 compared to non-affected or DMSO controls (as indicated).

Article Snippet: Male MPS I mice model [ ] ( Idua tm1.1Kmke allele homozygous, referred to as Idua − ; note we observed visibly stronger MPS I appearance in the male mice) and male and female MPS VI mice model [ , ] [ , ] ( Arsb m1J allele homozygous, referred to as Arsb − ) and wildtype (WT) littermates were obtained from internal breeding of stock mice derived from The Jackson Laboratory (MPS I model: strain No: 017681; B6.129S- Idua tm1.1Kmke /J and MPS VI model: strain No: 005598; C57BL/6 J- Arsb m1J /GrsrJ).

Techniques: Control, Fluorescence

Journal: Molecular Genetics and Metabolism Reports

Article Title: Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models

doi: 10.1016/j.ymgmr.2023.101011

Figure Lengend Snippet: GAG Quantification in Tissues from MPS VI and MPS I mice treated with odiparcil. A, B. CS, DS and HS quantification in extracts from liver (A) and eye (B) of WT control, Arsb- control and Arsb- mice treated with the indicated dose of odiparcil (g odiparcil per kg diet); C, D. CS, DS and HS quantification in extracts from liver (C) and eye (D) of WT control, Idua- control and Idua- mice treated with the indicated dose of odiparcil (g odiparcil per kg diet); Data is presented as mean ± SEM. Groups were statistically analysed using a one-way ANOVA followed by Dunnett's test; ** p < 0.01, *** p < 0.001 compared to WT or Arsb − or Idua- controls (as indicated).

Article Snippet: Male MPS I mice model [ ] ( Idua tm1.1Kmke allele homozygous, referred to as Idua − ; note we observed visibly stronger MPS I appearance in the male mice) and male and female MPS VI mice model [ , ] [ , ] ( Arsb m1J allele homozygous, referred to as Arsb − ) and wildtype (WT) littermates were obtained from internal breeding of stock mice derived from The Jackson Laboratory (MPS I model: strain No: 017681; B6.129S- Idua tm1.1Kmke /J and MPS VI model: strain No: 005598; C57BL/6 J- Arsb m1J /GrsrJ).

Techniques: Control

Journal: Orphanet Journal of Rare Diseases

Article Title: Registries for orphan drugs: generating evidence or marketing tools?

doi: 10.1186/s13023-020-01519-0

Figure Lengend Snippet: Overview of the LSD disease registries sponsored by pharmaceutical industry

Article Snippet: MPS VI , Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) , 2005 , BioMarin Pharmaceutical , 200.

Techniques: