- Grants since 2016
- 611
- Awarded, last 5 years (where amounts are published)
- €94.6M
- Funded works, last 5 years
- 5 837
- Funded works, all time
- 8 569
Grants
2024–2026 · grant
Investigating the role of PERM1 in transcriptional regulation of oxidative metabolism, in Duchenne Muscular Dystrophy.
Giuseppina Caretti · University of Milan
2024–2026 · grant
One gene, two diseases: The pathologic role of IGLV1-44 in AL amyloidosis and POEMS
Stefano Ricagno · University of Milan
2024–2027 · grant
Treating CMT2A: MFN1 Gene Enhancement as a Therapeutic Strategy
Stefania Corti · University of Milan
2024–2025 · grant
Characterization of the zebrafish SLC6A1 KO mutant line and identification of new pharmacological treatments in SLC6A1-associated neurodevelopment disorders
Andrea Vettori · University of Verona
2024–2026 · grant
FishCOLler: generation of in vivo models of COL4A1/A2 small vessel disease in tractable zebrafish embryos to unravel patho-mechanisms and targetable molecules
Antonella Lauri · Ospedale Pediatrico Bambino Gesù - Irccs
2024–2027 · grant
Bone health in adult patients with Duchenne Muscular Dystrophy (DMD). A survey on osteoporosis, fragility fractures and bone-targeted therapies
Chiara Panicucci · Irccs - Istituto Giannina Gaslini
2024–2026 · grant
Exploring the roles of autophagy regulation and mitochondria homeostasis in Okinawa-type HMSN, molecular mechanisms and clinical opportunities
Francesco Cecconi · Università Cattolica del Sacro Cuore
2024–2026 · grant
Investigating the relationship between HTT CAG repeats, somatic instability and neuronal dysfunction in Huntington's Disease using a highly parallel stem cell-based single cell approach.
Dario Besusso · University of Milan
2024–2026 · grant
Targeting Interleukin 17 during nontuberculous mycobacterial lung infection in cystic fibrosis
Nicola Lore · Vita-Salute San Raffaele University
2024–2026 · grant
Role of Negr1 signaling dysregulation in Fragile X and other rare syndromic forms of autism.
Laura Cancedda · Italian Institute of Technology
2024–2026 · grant
A multitask lentiviral vector for ex vivo gene therapy of Duchenne Muscular Dystrophy
Giulio Cossu · Vita-Salute San Raffaele University
2024–2026 · grant
Delivery of cytoplasmic HDAC4 to treat DMD
Viviana Moresi · National Research Council
2024–2026 · grant
Deciphering the role of kielin/chordin-like protein in hepcidin regulation and its relevance in Juvenile Hemochromatosis 2A
Alessia Pagani · Vita-Salute San Raffaele University
2024–2026 · grant
Functional characterization of the mitochondrial protein CCDC58 and its role in 3-methylglutaconic aciduria type 9
Saverio Marchi · Marche Polytechnic University
2024–2026 · grant
Cell therapy of the GLUT1-DS using directed GLUT1 engineered endothelial cell progenitors
Paolo Monti · Vita-Salute San Raffaele University
2024–2027 · grant
C1q inhibition in muscular dystrophy
Stefano Biressi · University of Trento
2024–2027 · grant
The interplay between Inborn Error of Immunity and blood disorders: unravelling immune defects behind common haematological diseases
Maria Teresa Voso · University of Rome Tor Vergata
2024–2026 · grant
Nav1.1 translation enhancement-based therapy to treat Dravet Syndrome
Gaia Colasante · Vita-Salute San Raffaele University
2024–2026 · grant
Development and application of spastin recovery therapeutic approaches in SPG4-HSP pre-clinical models
Cinzia Rinaldo · National Research Council
2024–2026 · grant
Evaluation of a new medical device shielding the skin of XP patients from UV-light
Antonio Fiorenzo Peverali · National Research Council
2024–2025 · grant
Alterations of SACSIN RNA-binding properties are connected to the development of ARSACS.
Roberto Giambruno · National Research Council
2024–2026 · grant
Role of Aquaporin-4 isoform mutations in megalencephalic leukoencephalopathy with subcortical cysts
Antonio Frigeri · University of Bari Aldo Moro
2024–2025 · grant
Pilot study for Unveiling ZNF865 role in undiagnosed rare neurodevelopmental disease (PUZZLE)
Michela Ori · University of Pisa
2024–2025 · grant
Understanding the mechanism and pathological consequences of mutations on the developmentally regulated SCN8A alternative splicing of exon 5.
Marco Baralle · International Centre for Genetic Engineering and Biotechnology
2024–2026 · grant
Genetic, cellular, and structural basis of the neurodegenerative pathology FENIB
Maria Elena Miranda Banos · Sapienza University of Rome
2024–2026 · grant
Translational correction approaches targeting nonsense mutations in hemophilia A (TranslEight)
Alessio Branchini · University of Ferrara
2024–2026 · grant
Cell Free DNA
Daniela Cesana · San Raffaele Telethon Institute For Gene Therapy - Sr-tiget
2024–2026 · grant
A Drosophila model of Type B Kufs/CLN13 disease to study pathogenesis and Cathepsin F function
Thomas Vaccari · University of Milan
2024–2026 · grant
Unraveling the role of the putative ATPase ATP5MGL in beta-thalassemia
Antonella Nai · Vita-Salute San Raffaele University
2024–2026 · grant
Characterization of a new therapeutic strategy for Phelan McDermid syndrome by rescuing dysregulated protein synthesis
Chiara Verpelli · National Research Council
2024–2026 · grant
Role of ferroptosis in β-talassemia pathogenesis
Francesca Carlomagno · University of Naples Federico II
2024–2027 · grant
Sphingolipid metabolic imbalance in murine and hiPSC RTT models: development of a drug repurposing-based therapy
Floriana Della Ragione · National Research Council
2024–2026 · grant
Evaluation of new protease inhibitors in vitro and animal models as antivirulence therapy against Pseudomonas aeruginosa infections of Primary Ciliary Dyskinesia patients
Giuseppantonio Maisetta · University of Pisa
2024–2026 · grant
Gait abnormalities in tuberous sclerosis complex: a clinical and neurophysiologic study
Romina Moavero · Ospedale Pediatrico Bambino Gesù - Irccs
2024–2025 · grant
Investigating the interplay between ACVR1/Alk2 signalling and autophagy in Fibrodysplasia Ossificans Progressiva (FOP)
Venturina Stagni · National Research Council
2024–2029 · grant
STOP-HSP.net: Storing a collection Of Patients with HSP – network
Filippo Maria Santorelli · Fondazione Stella Maris - Irccs
2024–2026 · grant
Investigating the brain circuit and molecular alterations associated with the visuomotor integration function in CDKL5 zebrafish mutant
Nicola Facchinello · National Research Council
2024–2026 · grant
DeveLopment of a pharmacologIcal neonatal treatment and an in uterO fetal gene therapy for leigh syNdrome - LION
Dario Brunetti · Fondazione Istituto Neurologico Carlo Besta - Irccs
2024–2025 · grant
New molecules for the treatment of Sanfilippo Syndrome
Elvira De Leonibus · Telethon Institute Of Genetics And Medicine (tigem)
2024–2026 · grant
Dissecting the function of PRR12 and its role in Neuroocular Syndrome through a multi-omic approach
Giovanni Perini · University of Bologna