Pubblicazioni

Revolutionizing CAR T-Cell Therapies: Innovations in Genetic Engineering and Manufacturing to Enhance Efficacy and Accessibility

2024 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Giorgioni, Lorenzo; Ambrosone, Alessandra; Cometa, Maria Francesca Paola; Salvati, Anna Laura; NisticÃ², Robert; Magrelli, Armando

Unveiling the Future of Cardiac Care: A Review of Gene Therapy in Cardiomyopathies

2024 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Venturiello, Damiano; Tiberi, Pier Giorgio; Perulli, Francesco; Nardoianni, Giulia; Guida, Leonardo; Barsali, Carlo; Terrone, Carlo; Cianca, Alessandro; Lustri, Camilla; Sclafani, Matteo; Tini, Giacomo; Barbato, Emanuele; Musumeci, Beatrice

Nano-immunomodulation: a new strategy for skeletal muscle diseases and aging?

2023 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Millozzi, Francesco; Papait, Andrea; Bouche', Marina; Parolini, Ornella; Palacios, Daniela

CRISPR/Cas9 and piggyBac Transposon-Based Conversion of a Pathogenic Biallelic TBCD Variant in a Patient-Derived iPSC Line Allows Correction of PEBAT-Related Endophenotypes

2023 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Muto, Valentina; Benigni, Federica; Magliocca, Valentina; Borghi, Rossella; Flex, Elisabetta; Pallottini, Valentina; Rosa, Alessandro; Compagnucci, Claudia; Tartaglia, Marco

Human iPSC model reveals a central role for NOX4 and oxidative stress in Duchenne cardiomyopathy

2022 - STEM CELL REPORTS

Duelen, R.; Costamagna, D.; Gilbert, G.; Waele, L. D.; Goemans, N.; Desloovere, K.; Verfaillie, C. M.; Sipido, K. R.; Buyse, G. M.; Sampaolesi, M.

Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

2022 - MOLECULAR THERAPY NUCLEIC ACIDS

Cardinali, B.; Provenzano, C.; Izzo, M.; Voellenkle, C.; Battistini, J.; Strimpakos, G.; Golini, E.; Mandillo, S.; Scavizzi, F.; Raspa, M.; Perfetti, A.; Baci, D.; Lazarevic, D.; Garcia-Manteiga, J. M.; Gourdon, G.; Martelli, F.; Falcone, G.

Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing

2022 - INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Izzo, Mariapaola; Battistini, Jonathan Isacco; Provenzano, Claudia; Martelli, Fabio; Cardinali, Beatrice; Falcone, Germana

Commentary â CRISPR-based techniques: Cas9, cas13 and their applications in the era of COVID-19

2021 - EUROPEAN REVIEW FOR MEDICAL AND PHARMACOLOGICAL SCIENCES

Zaami, S.; Piergentili, R.; Marinelli, E.; Montanari Vergallo, G.

Comment on the article by Zaami S, et al. âCRISPR-based techniques: Cas9, Cas13 and their applications in the era of COVID-19â. Eur Rev Med Pharmacol Sci 2021; 25 (3): 1752-1761

2021 - EUROPEAN REVIEW FOR MEDICAL AND PHARMACOLOGICAL SCIENCES

Del Rio, Alessandro; Negro, Francesca; Umani Ronchi, Federica

Thalassemias. From gene to therapy

2021 - MOLECULAR ASPECTS OF MEDICINE

De Simone, G.; Quattrocchi, A.; Mancini, B.; di Masi, A.; Nervi, C.; Ascenzi, P.

Anti-ageing gene therapy. Not so far away?

2019 - AGEING RESEARCH REVIEWS

Vaiserman, A.; De Falco, E.; Koliada, A.; Maslova, O.; Balistreri, C. R.

Stimulation of CRISPR-mediated homology-directed repair by an engineered RAD18 variant

2019 - NATURE COMMUNICATIONS

Nambiar, T. S.; Billon, P.; Diedenhofen, G.; Hayward, S. B.; Taglialatela, A.; Cai, K.; Huang, J. -W.; Leuzzi, G.; Cuella-Martin, R.; Palacios, A.; Gupta, A.; Egli, D.; Ciccia, A.

Molecular imaging of human embryonic stem cells stably expressing human PET reporter genes after zinc finger nuclease-mediated genome editing

2017 - THE JOURNAL OF NUCLEAR MEDICINE

Wolfs, Esther; Holvoet, Bryan; Ordovas, Laura; Breuls, Natacha; Helsen, Nicky; SchÃ¶nberger, Matthias; Raitano, Susanna; Struys, Tom; Vanbilloen, Bert; Casteels, Cindy; Sampaolesi, Maurilio; Van Laere, Koen; Lambrichts, Ivo; Verfaillie, Catherine M.; Deroose, Christophe M.