Models and therapeutic approaches for Niemann-Pick (A/B and C) and other lysosomal storage disorders 

Gomez Grau, Marta (Date of defense: 2015-12-18)

Lysosomal storage disorders (LSDs) are a group of more than 50 different genetic disorders due to the lack of degradation of substrates within lysosomes. Most of them are caused by mutations in genes coding for lysosomal ...