SMABEYOND Viero (DFM.AD004.274)

Area tematica

Scienze fisiche e tecnologie della materia

Area progettuale

Struttura responsabile del progetto di ricerca

Istituto di biofisica (IBF)

Responsabile di progetto

GABRIELLA VIERO
Telefono: 0461 314033
E-mail: gabriella.viero@gmail.com

Abstract

The project analyzes intrinsic defects in peripheral organs (WP1), evaluate the organ specific molecular and cellular functions of the SMN protein in relevant organs (WP2), and translate these findings to SMA patient derived models, which we will treat with a systemic SMA drug currently under clinical evaluation (WP3). The SMA field involves stakeholders, which allow early stage researchers to personally interact with basic scientists, clinicians, pharmaceutical companies and patient organizations. For our training network, we will combine this vertical integration with a broad perspective on multiple organ systems in SMA. The training strategy assures career options and employability of early stage researchers beyond the SMA field. We will go beyond the motoneuron and identify organs, mechanisms and molecules that could be targets for the peripheral aspects of SMA.

Data inizio attività

01/10/2020

Parole chiave

Spinal Muscular Atrophy, motoneuron degeneration

Ultimo aggiornamento: 12/12/2024