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Functional Genomics of the Lysosome

Total Cost €


EC-Contrib. €






 LYSOSOMICS project word cloud

Explore the words cloud of the LYSOSOMICS project. It provides you a very rough idea of what is the project "LYSOSOMICS" about.

genes    potentially    phenotypic    content    plan    normal    lysosome    model    lot    learned    storage    challenged    transcriptional    pathological    time    subject    perturbations    degradation    severe    lysosomal    cell    acts    newly    diseases    investigation    genetic    relevance    group    onset    static    collection    computational    neurodegenerative    biology    generate    viewed    lysosomics    adapt    regulate    recycling    global    neurodegeneration    crispr    organellar    mice    performed    health    mostly    hub    suggesting    regulation    parkinson    cas9    environmental    predict    waste    fret    gain    strategies    dysfunction    lines    routine    purposes    protein    for    interactions    exploitable    validated    mechanisms    organelle    discover    therapeutic    view    inherited    pertaining    entirely    medaka    assays    function    homeostasis    perform    types    cellular    omics    signalling    disorders    performs    alzheimer    vivo    opened    clues    physiological    gene    models    disease   

Project "LYSOSOMICS" data sheet

The following table provides information about the project.


Organization address
address: VIA VARESE 16/B
city: ROMA
postcode: 185

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Italy [IT]
 Total cost 2˙362˙562 €
 EC max contribution 2˙362˙562 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2015-AdG
 Funding Scheme ERC-ADG
 Starting year 2016
 Duration (year-month-day) from 2016-10-01   to  2021-09-30


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    FONDAZIONE TELETHON IT (ROMA) coordinator 2˙362˙562.00


 Project objective

For a long time the lysosome has been viewed as a “static” organelle that performs “routine” work for the cell, mostly pertaining to degradation and recycling of cellular waste. My group has challenged this view and used a systems biology approach to discover that the lysosome is subject to a global transcriptional regulation, is able to adapt to environmental clues, and acts as a signalling hub to regulate cell homeostasis. Furthermore, an emerging role of the lysosome has been identified in many types of diseases, including the common neurodegenerative disorders Parkinson's and Alzheimer’s. These findings have opened entirely new fields of investigation on lysosomal biology, suggesting that there is a lot to be learned on the role of the lysosome in health and disease. The goal of LYSOSOMICS is to use “omics” approaches to study lysosomal function and its regulation in normal and pathological conditions. In this “organellar systems biology project” we plan to perform several types of genetic perturbations in three widely used cell lines and study their effects on lysosomal function using a set of newly developed cellular phenotypic assays. Moreover, we plan to identify lysosomal protein-protein interactions using a novel High Content FRET-based approach. Finally, we will use the CRISPR-Cas9 technology to generate a collection of cellular models for all lysosomal storage diseases, a group of severe inherited diseases often associated with early onset neurodegeneration. State-of-the-art computational approaches will be used to predict gene function and identify disease mechanisms potentially exploitable for therapeutic purposes. The physiological relevance of newly identified pathways will be validated by in vivo studies performed on selected genes by using medaka and mice as model systems. This study will allow us to gain a comprehensive understanding of lysosomal function and dysfunction and to use this knowledge to develop new therapeutic strategies.


year authors and title journal last update
List of publications.
2018 Rosa Puertollano, Shawn M Ferguson, James Brugarolas, Andrea Ballabio
The complex relationship between TFEB transcription factor phosphorylation and subcellular localization
published pages: e98804, ISSN: 0261-4189, DOI: 10.15252/embj.201798804
The EMBO Journal 2019-06-13
2017 Chiara Di Malta, Diletta Siciliano, Alessia Calcagni, Jlenia Monfregola, Simona Punzi, Nunzia Pastore, Andrea N. Eastes, Oliver Davis, Rossella De Cegli, Angela Zampelli, Luca G. Di Giovannantonio, Edoardo Nusco, Nick Platt, Alessandro Guida, Margret Helga Ogmundsdottir, Luisa Lanfrancone, Rushika M. Perera, Roberto Zoncu, Pier Giuseppe Pelicci, Carmine Settembre, Andrea Ballabio
Transcriptional activation of RagD GTPase controls mTORC1 and promotes cancer growth
published pages: 1188-1192, ISSN: 0036-8075, DOI: 10.1126/science.aag2553
Science 356/6343 2019-08-05
2019 Gabriella Doronzo, Elena Astanina, Davide Corà, Giulia Chiabotto, Valentina Comunanza, Alessio Noghero, Francesco Neri, Alberto Puliafito, Luca Primo, Carmine Spampanato, Carmine Settembre, Andrea Ballabio, Giovanni Camussi, Salvatore Oliviero, Federico Bussolino
TFEB controls vascular development by regulating the proliferation of endothelial cells
published pages: e98250, ISSN: 0261-4189, DOI: 10.15252/embj.201798250
The EMBO Journal 38/3 2019-08-05
2018 Gennaro Napolitano, Alessandra Esposito, Heejun Choi, Maria Matarese, Valerio Benedetti, Chiara Di Malta, Jlenia Monfregola, Diego Luis Medina, Jennifer Lippincott-Schwartz, Andrea Ballabio
mTOR-dependent phosphorylation controls TFEB nuclear export
published pages: , ISSN: 2041-1723, DOI: 10.1038/s41467-018-05862-6
Nature Communications 9/1 2019-08-05

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