Stem Cell Biology and Pharmacology of Neurodegenerative Diseases

The laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases, led by Prof. Elena Cattaneo, is part of the Department of Bioscience at the University of Milan. The lab has relocated in INGM in July 2015, on the basis of a collaborative agreement between the University of Milan and INGM.

The laboratory studies Huntington’s Disease (HD), a rare genetic and neurodegenerative disease. The goal is to understand the physiopathology of this disease, in order to develop pharmacological, genetic and cellular strategies that could slow down the course of the illness or prevent its onset.

Currently, the laboratory is pursuing the following strategies:

  • Investigate Huntingtin function in adulthood and throughout evolution (collaboration with Bioinformatic Unit – Dr Riccardo Rossi).
  • Define the transcriptional profile of the developing human ventral telencephalon (giving rise to the striatum) at the single cell level to uncover regional/cell type-specific and time controlled expression of coding and non-coding transcripts that are involved in striatal differentiation in healthy and HD individuals (collaboration with Prof. Pagani’s Group);
  • Investigate the differentiation potential of human pluripotent stem cells carrying the mutant Huntingtin to pinpoint the pathophysiological mechanisms underlying HD.
  • Establish new high throughput screening methods to characterize cell identity during in vitro differentiation of human pluripotent stem cells to authentic striatal neurons, in order to optimize our striatal differentiation protocols.

The laboratory works with Huntington’s disease clinical experts in Italy and abroad, to assess the diagnostic and therapeutic potential of our research findings for clinical translation and application.

The laboratory is composed of one full professor, one associate professor, one university researcher, Post-Docs, PhD Students, Junior Fellows and Master Students, all of them are part of the University of Milan.


  • Molecular Profiling of the developing human striatum.
  • Optimization of striatal differentiation protocols using human pluripotent stem cells.
  • Pathogenic mechanisms in Huntington’s Disease.
  • Huntingtin function in adulthood and throughout evolution.
  • Cholesterol delivery in the HD brain.


Mattia BattistellaPhD;
Dario BesussoSenior;;
Ilaria CampusResearch;
Paola ConfortiSenior;
Gianluca DamaggioResearch;
Maura GalimbertiPost;
Raffaele IennacoPost;
Veronica MarroccoliJunior;
Maria Lidia MignognaPost;
Davide ScalzoJunior;
Linda ScaramuzzaPost;
Andrea ScolzResearch;
Tiziana TurrisiLaboratory Assistant and Grant;
Rafael Ugarte ChaconProject;
Michela VillaPhD;
Martina ZobelPost;
Chiara Zuccato


Elena Cattaneo has published >160 papers in peer-reviewed journals (including Science, Nature, Nature Genetics, Nature Neuroscience, Journal of Neuroscience, JBC). Her H-index is 64.

Selection of the 20 most relevant peer-reviewed publications (from 2000):