| Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase F Puzzo, P Colella, MG Biferi, D Bali, NK Paulk, P Vidal, F Collaud, ... Science translational medicine 9 (418), eaam6375, 2017 | 123 | 2017 |
| AAV gene transfer with tandem promoter design prevents anti-transgene immunity and provides persistent efficacy in neonate pompe mice P Colella, P Sellier, HC Verdera, F Puzzo, L van Wittenberghe, ... Molecular Therapy-Methods & Clinical Development 12, 85-101, 2019 | 76 | 2019 |
| Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors M Hösel, A Huber, S Bohlen, J Lucifora, G Ronzitti, F Puzzo, F Boisgerault, ... Hepatology 66 (1), 252-265, 2017 | 46 | 2017 |
| Rescue of GSDIII phenotype with gene transfer requires liver-and muscle-targeted GDE expression P Vidal, S Pagliarani, P Colella, HC Verdera, L Jauze, M Gjorgjieva, ... Molecular Therapy 26 (3), 890-901, 2018 | 36 | 2018 |
| Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients E Masat, P Laforêt, M De Antonio, G Corre, B Perniconi, N Taouagh, ... Scientific reports 6 (1), 36182, 2016 | 34 | 2016 |
| Rescue of advanced Pompe disease in mice with hepatic expression of secretable acid α-glucosidase U Cagin, F Puzzo, MJ Gomez, M Moya-Nilges, P Sellier, C Abad, ... Molecular Therapy 28 (9), 2056-2072, 2020 | 20 | 2020 |
| Aptamer-programmable adeno-associated viral vectors as a novel platform for cell-specific gene transfer F Puzzo, C Zhang, BP Gray, F Zhang, BA Sullenger, MA Kay Molecular Therapy-Nucleic Acids 31, 383-397, 2023 | 13 | 2023 |
| Targeting mitochondrial defects to increase longevity in animal models of neurodegenerative diseases E Casajus Pelegay, F Puzzo, A Yilmazer, U Cagin Reviews on Biomarker Studies of Metabolic and Metabolism-Related Disorders …, 2019 | 12 | 2019 |
| Improved genome editing through inhibition of FANCM and members of the BTR dissolvase complex G de Alencastro, F Puzzo, M Pavel-Dinu, F Zhang, S Pillay, K Majzoub, ... Molecular Therapy 29 (3), 1016-1027, 2021 | 11 | 2021 |
| Acid-alpha glucosidase variants and uses thereof F Mingozzi, G Ronzitti, P Colella, F Puzzo US Patent App. 17/072,065, 2021 | 3 | 2021 |
| O. 6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease S Armour, J Nordin, HC Verdera, D Cohen, P Sellier, M Crosariol, ... Neuromuscular Disorders 29, S39, 2019 | 3 | 2019 |
| Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease SM Armour, J Nordin, F Puzzo, U Cagin, M Crosariol, F Collaud, ... Mol Genet Metab 126, S24, 2019 | 2 | 2019 |
| Correction of Advanced Pompe Phenotype in Mice with AAV Liver Gene Transfer of Secretable GAA F Puzzo, U Cagin, M Moya-Nilges, L Van Wittenberghe, N Daniele, ... MOLECULAR THERAPY 26 (5), 312-312, 2018 | 2 | 2018 |
| Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes P Colella, F Puzzo, M Biferi, D Bali, N Paulk, P Vidal, F Collaud, ... Neuromuscular Disorders 27, S246, 2017 | 2 | 2017 |
| ESGCT XXV anniversary congress in collaboration with the German Society for Gene Therapy, October 17–20, 2017 Berlin, Germany S Maffioletti Human Gene Therapy 28 (12), A1-A125, 2017 | 2 | 2017 |
| Chimeric polypeptides and uses thereof P Colella, F Puzzo, F Mingozzi US Patent App. 17/770,641, 2023 | 1 | 2023 |
| Tandem promoter design confers tolerogenic and persistent transgene expression to AAV gene therapy in neonate Pompe mice P Colella, P Sellier, H Costa-Verdera, F Puzzo, L van Wittenberghe, ... HUMAN GENE THERAPY 29 (12), A166-A166, 2018 | 1 | 2018 |
| AAV-Mediated Liver Expression of Secretable GAA Shows Therapeutic Efficacy in Pompe Mice at Low Vector Doses F Puzzo, U Cagin, L Van Wittenberghe, N Daniele, N Guerchet, P Colella, ... MOLECULAR THERAPY 26 (5), 386-386, 2018 | 1 | 2018 |
| Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease P Vidal, G Ronzitti, F Collaud, MS Sola, P Collela, F Puzzo, HC Verdera, ... Neuromuscular Disorders 27, S246, 2017 | 1 | 2017 |
| AAV-mediated genome editing is influenced by the formation of R-loops F Puzzo, MP Crossley, A Goswami, F Zhang, K Pekrun, JL Garzon, ... bioRxiv, 2024 | | 2024 |
Pasqualina (Lina) ColellaStanford University在 stanford.edu 的电子邮件经过验证
