CORDIS Project
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This project investigates Spinal Muscular Atrophy (SMA) beyond motoneurons, focusing on the role of the Survival of Motoneuron protein in peripheral organs. It aims to identify new therapeutic targets for SMA treatment across multiple organ systems.
Spinal Muscular Atrophy (SMA) is a monogenic motoneuron disease with a neuromuscular phenotype resulting in infant death in severe cases.
Besides motoneurons in the central nervous system (CNS), there is growing evidence of an involvement of peripheral organs. SMA is caused by reduced Survival of Motoneuron (SMN) protein levels and SMN is ubiquitously expressed.
Therefore, SMA patients show reduced SMN levels also in peripheral organs. A restoration of SMN levels in the CNS is a potent therapeut…
SMATHERIA GGMBH
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Netherlands, Utrecht
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
STIFTUNG TIERAERZTLICHE HOCHSCHULE HANNOVER
Germany, Hannover
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
KLINIKUM DER UNIVERSITAET ZU KOELN
Germany, Koeln
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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