Experimental models

Area

Endocrine, Nutritional and Metabolic Diseases

Indication

Sanfilippo Syndrome B and C

System

In vitro

Model references

Stem Cell Reports. 2015; 5: 546-57

Sanfilippo C IPSC neurons and astrocytes: patient-derived IPCs fibroblasts of Sanfilippo C syndrome patients differentiation into neu- rons

Applications

  • Cell reprogramming.
  • Mutant alleles characterization and expression.
  • Study of mechanisms that generate recombinant alleles traffic.
  • Nonsense mediated decay genotype-phenotype correlation gene silencing.
  • New therapeutic strategies research.
  • Splicing analysis (microexon).

Principal investigators

Daniel-Raul Grinberg, Ma Lluïsa Vilageliu.

Other group publications

PubMed publications.

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