| Fetal gene therapy for neurodegenerative disease of infants G Massaro, CNZ Mattar, AMS Wong, E Sirka, SMK Buckley, BR Herbert, ... Nature medicine 24 (9), 1317-1323, 2018 | 148 | 2018 |
| Epilepsy gene therapy using an engineered potassium channel A Snowball, E Chabrol, RC Wykes, T Shekh-Ahmad, JH Cornford, A Lieb, ... Journal of Neuroscience 39 (16), 3159-3169, 2019 | 91 | 2019 |
| AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease MP Hughes, DA Smith, L Morris, C Fletcher, A Colaco, M Huebecker, ... Human molecular genetics 27 (17), 3079-3098, 2018 | 71 | 2018 |
| Delineating pathological pathways in a chemically induced mouse model of Gaucher disease A Vardi, H Zigdon, A Meshcheriakova, AD Klein, C Yaacobi, R Eilam, ... The Journal of pathology 239 (4), 496-509, 2016 | 66 | 2016 |
| Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development G Massaro, AF Geard, W Liu, O Coombe-Tennant, SN Waddington, ... Biomolecules 11 (4), 611, 2021 | 35 | 2021 |
| Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload N Plotegher, D Perocheau, R Ferrazza, G Massaro, G Bhosale, F Zambon, ... Cell Death & Differentiation, 1-1, 2020 | 34* | 2020 |
| Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes G Massaro, MP Hughes, SM Whaler, KL Wallom, DA Priestman, FM Platt, ... Human Molecular Genetics, 2020 | 31 | 2020 |
| Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload N Plotegher, D Perocheau, R Ferrazza, G Massaro, G Bhosale, F Zambon, ... Cell Death & Differentiation, 2019 | 31 | 2019 |
| Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease SM kleine Holthaus, S Martin-Herranz, G Massaro, M Aristorena, J Hoke, ... bioRxiv, 673848, 2019 | 26 | 2019 |
| Perinatal systemic gene delivery using adeno-associated viral vectors R Karda, SMK Buckley, CN Mattar, J Ng, G Massaro, MP Hughes, ... Frontiers in molecular neuroscience 7, 89, 2014 | 23 | 2014 |
| Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective A Di Donfrancesco, G Massaro, I Di Meo, V Tiranti, E Bottani, D Brunetti Pharmaceutics 14 (6), 1287, 2022 | 16 | 2022 |
| A GLP1 receptor agonist diabetes drug ameliorates neurodegeneration in a mouse model of infantile neurometabolic disease L Poupon-Bejuit, MP Hughes, W Liu, A Geard, N Faour-Slika, S Whaler, ... Scientific Reports 12 (1), 1-17, 2022 | 9 | 2022 |
| A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease MP Hughes, HR Nelvagal, O Coombe-Tennant, D Smith, C Smith, ... Cells 12 (12), 1619, 2023 | 5 | 2023 |
| Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors Z Whiteley, G Massaro, G Gkogkos, A Gavriilidis, SN Waddington, ... Nanoscale 15 (12), 5865-5876, 2023 | 4 | 2023 |
| PPAR-gamma agonist pioglitazone recovers mitochondrial quality control in fibroblasts from PITRM1-deficient patients A Di Donfrancesco, C Berlingieri, M Giacomello, C Frascarelli, ... Frontiers in Pharmacology 14, 2023 | 2 | 2023 |
| Engineering an Autonucleolytic Mammalian Suspension Host Cell Line to Reduce DNA Impurity Levels in Serum-Free Lentiviral Process Streams G Howe, M Wasmuth, P Emanuelle, G Massaro, AA Rahim, S Ali, ... ACS Synthetic Biology, 2024 | 1 | 2024 |
| Intravenously administered gene therapy for neuronopathic Gaucher disease G Massaro UCL (University College London), 2018 | 1 | 2018 |
| Improving the efficiency of AAV gene delivery vectors via capsid design and bioprocess engineering L Hall, G Massaro, A Rahim, P Majumder, Q Rafiq HUMAN GENE THERAPY 33 (23-24), A40-A40, 2022 | | 2022 |
| AAV9-mediated gene therapy in a knock-in mouse model of infantile neuroaxonal dystrophy S Whaler, AF Geard, L Poupon-Bejuit, G Massaro, MP Hughes, K Lalji, ... HUMAN GENE THERAPY 33 (23-24), A12-A12, 2022 | | 2022 |
| Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease S Herranz-Martin, G Massaro, M Aristorena, J Hoke, MP Hughes, ... Human Molecular Genetics, 2019 | | 2019 |
