- 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
2021–2022 · grant
mRNA replacement therapy for GSD-Ib
Nicola Brunetti-Pierri · Telethon Institute Of Genetics And Medicine (tigem)
2021–2025 · grant
Dissecting new functions of the Nijmegen breakage syndrome gene in cerebellar development
Giuseppe Giannini · Sapienza University of Rome
2021–2025 · grant
Treating cystic fibrosis with a competing peptide targeting PI3Kγ scaffold activity
Alessandra Ghigo · University of Turin
2021–2024 · grant
3D modelling of rare muscular diseases, a powerful platform for basic studies and drug validation
Dorianna Sandonà · University of Padua
2021–2023 · grant
MAMA: Molecular Analysis and manipulation of Metabolic signalling in Adenylosuccinase deficiency
Francesco Cecconi · University of Rome Tor Vergata
2021–2025 · grant
Insight CLN5: Approaching therapies in the neuronal ceroid lipofuscinosis, using Zebrafish as a Tool
Maria Marchese · Fondazione Stella Maris - Irccs
2021–2024 · grant
Allele-specific CRISPR- engineered Cpf1 genome editing to treat ocular surface disorder in ectrodactyly–ectodermal dysplasia–clefting (EEC) syndrome
Michele De Luca · Università Degli Studi Di Modena E Reggio Emilia Unimore
2021–2025 · grant
Exploit iron-burden astrocytes and mouse models to define the therapy for PKAN and CoPAN.
Sonia Levi · Vita-Salute San Raffaele University
2021–2022 · grant
EVALUATION OF THERAPEUTIC STRATEGIES TO CORRECT THE BASIC DEFECT IN PRIMARY CILIARY DYSKINESIA
Luis Juan Vicente Galietta · Telethon Institute Of Genetics And Medicine (tigem)
2021–2024 · grant
Cell-Penetrating SIL1 Protein Replacement Therapy for Marinesco-Sjogren Syndrome
Michele Sallese · University of Chieti-Pescara
2021–2022 · grant
Artificial Intelligence for individual profiling and prediction: probing the FractaL dimension of brain MRI in FriEdreich ataXia and SCAs using the ENIGMA-Ataxia international meta-dataset (FLEX-AI)
Stefano Diciotti · University of Bologna
2021–2024 · grant
Novel therapeutic approaches for AEC syndrome
Caterina Missero · University of Naples Federico II
2021–2024 · grant
GABAA-receptor defects in CDKL5 deficiency disorder: molecular mechanisms and targeting by synthetic neuroactive steroids
Charlotte Kilstrup-Nielsen · University of Insubria
2021–2023 · grant
Preclinical efficacy study of PERK signaling inhibitors and TUDCA in Marinesco-Sjögren syndrome
Roberto Chiesa · Mario Negri Institute for Pharmacological Research
2021–2024 · grant
Modulation of ADAM10 at the pre- and post-synaptic terminal and its contribution in Huntington's Disease cortico-striatal dysfunction
Chiara Zuccato · University of Milan
2021–2024 · grant
Modeling Wolman disease using genetically engineered human liver organoids
Giovanni Sorrentino · International Centre for Genetic Engineering and Biotechnology
2021–2023 · grant
Searching new molecular targets in Fibrodysplasia Ossificans Progressiva (FOP): is the autophagy signalling a good candidate?
Venturina Stagni · National Research Council
2021–2025 · grant
UBIAD1 and ferroptosis: exploring a cure for Schnyder Corneal Dystrophy (SCD)
Massimo Santoro · Fondazione Per La Ricerca Biomedica Avanzata Onlus - Istituto Veneto Di Medicina Molecolare (vimm)
2020–2024 · grant
Store-Operated Calcium Entry (SOCE): role in skeletal muscle function and disease
Feliciano Protasi · University of Chieti-Pescara
2020–2022 · grant
Deciphering dysfunctional metabolic pathways in nephropatic cystinosis
Gennaro Napolitano · Telethon Institute Of Genetics And Medicine (tigem)
2020–2024 · grant
New insights on the pathogenesis of hereditary Cerebral Cavernous Malformations
Massimiliano Pagani · Ifom Ets - Istituto Fondazione Di Oncologia Molecolare
2020–2024 · grant
Improving developmental trajectories in 22q11.2 deletion syndrome by oxytocin: focus on anti-inflammatory effects
Francesco Papaleo · Italian Institute of Technology
2020–2023 · grant
Mechanisms of axonal degeneration in late onset CMT1B neuropathies: molecular pathways and therapeutic approaches
Maurizio D'Antonio · Fondazione Centro San Raffaele
2020–2022 · grant
Multiplexed 3Cs identifies novel genetic interactions in Glut1 deficiency syndrome
Paolo Grumati · Telethon Institute Of Genetics And Medicine (tigem)
2020–2021 · grant
Investigating and harnessing cell intrinsic innate immunity against SARS-CoV-2 – Insight from the Aicardi-Goutières Syndrome
Anna Christina Kajaste-Rudnitski · San Raffaele Telethon Institute For Gene Therapy - Sr-tiget
2020–2022 · grant
AHF facility (Hystology)
Nicolina Cristina Sorrentino · Telethon Institute Of Genetics And Medicine (tigem)
2020–2023 · grant
Exploiting a bacterial redox cycler against mitochondrial diseases linked to respiratory complex dysfunction
Ildiko Szabo · University of Padua
2020–2023 · grant
TRIAL READINESS AND ENDPOINT ASSESSMENT IN CONGENITAL AND CHILDHOOD MYOTONIC DYSTROPHY
Valeria Sansone · Centro Clinico Nemo
2020–2024 · grant
INNOVATIVE STRATEGY TO ENHANCE THE EFFICIENCY AND SAFETY OF GENE THERAPY FOR CDKL5 DEFICENCY DISORDER
Elisabetta Ciani · University of Bologna
2020–2023 · grant
Exploiting regulatory T-cell metabolic reprogramming and vascular tropism as therapeutic tools for Familial Hypercholesterolaemia.
Giuseppe Danilo Norata · University of Milan
2020–2021 · grant
GLUT1 DEFICIENCY: NEW THERAPEUTIC STRATEGIES TO INCREASE GLUCOSE TRANSPORT ACROSS THE BLOOD BRAIN BARRIER (BBB)
Federico Zara · Irccs - Istituto Giannina Gaslini
2020–2022 · grant
Inactivation of the Niemann Pick C1 protein as a strategy to inhibit SARS-CoV2 infection
Maria Teresa Fiorenza · Sapienza University of Rome
2020–2024 · grant
Creatine Deficiency Syndrome: novel insight into brain function and therapeutic strategies
Laura Baroncelli · Fondazione Stella Maris - Irccs
2020–2022 · grant
Meninges as an overlooked pharmacological target for Globoid Cell Leukodystrophy
Francesco Bifari · University of Milan
2020–2022 · grant
Treatment strategy with the SGLT2 inhibitor in a GSD1b mouse model to correct metabolite-repair deficiency affecting neutrophils and kidney function
Francesco Trepiccione · Università Della Campania Ex Seconda Università Di Napoli
2020–2023 · grant
Regulation of pathogen-specific T-cell responses in patients with Hyper-IgE syndrome (HIES)
Jens Geginat · Istituto Nazionale Genetica Molecolare
2020–2024 · grant
From coagulation to angiogenesis: new roles for FVIII in endothelial functionality
Antonia Follenzi · Università degli Studi del Piemonte Orientale “Amedeo Avogadro”
2020–2022 · grant
Targeting mitochondrial metabolism to promote full neural development in Allan Herndon Dudley syndrome
Ilaria Decimo · University of Verona
2020–2022 · grant
Kidney Organoids for the Modelling of inherited renal diseases
Leopoldo Staiano · Telethon Institute Of Genetics And Medicine (tigem)
2020–2024 · grant
Structural and functional characterization of HERG potassium channels’ enhancers as a novel therapeutic strategy for Long QT Syndrome
Silvia Priori · Fondazione Salvatore Maugeri