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 | 85 | 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 | 67 | 2018 |
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer J Baruteau, DP Perocheau, J Hanley, M Lorvellec, E Rocha-Ferreira, ... Nature Communications 9 (1), 3505, 2018 | 44 | 2018 |
Immediate remote ischemic postconditioning reduces brain nitrotyrosine formation in a piglet asphyxia model E Rocha-Ferreira, B Rudge, MP Hughes, AA Rahim, M Hristova, ... Oxidative medicine and cellular longevity 2016, 2016 | 34 | 2016 |
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 29 (12), 1933-1949, 2020 | 29 | 2020 |
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease SM kleine Holthaus, S Herranz-Martin, G Massaro, M Aristorena, J Hoke, ... Human molecular genetics 28 (23), 3867-3879, 2019 | 27 | 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 | 22 | 2014 |
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), 13825, 2022 | 6 | 2022 |
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors R Karda, AA Rahim, AMS Wong, N Suff, JA Diaz, DP Perocheau, M Tijani, ... Scientific reports 10 (1), 2121, 2020 | 4 | 2020 |
Dipolar Bose-Hubbard model in finite-size real-space cylindrical lattices M Hughes, D Jaksch Physical Review A 105 (5), 053301, 2022 | 3 | 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 | 2 | 2023 |
305. Generation of light-producing, somatic-transgenic mice using lentivirus and adeno-associated virus vectors R Karda, DP Perocheau, SMK Buckley, JMKM Delhove, M Hughes, ... Molecular Therapy 24, S123, 2016 | 2 | 2016 |
Generation of light-producing somatic-transgenic mice using adeno-associated virus vector R Karda, AA Rahim, N Suff, JA Diaz, DP Perocheau, NP Martin, ... Molecular Therapy 26 (5), 40-40, 2018 | 1 | 2018 |
Development of gene therapy for Niemann-Pick Type C disease M Hughes, D Smith, L Morris, J Tordo, N Palomar-Martin, E Henckaerts, ... Human Gene Therapy 27 (7), A14-A14, 2016 | 1 | 2016 |
Gene therapy of niemann-pick disease type c M Hughes, A Rahim US Patent App. 17/910,686, 2023 | | 2023 |
Dysregulated Wnt and NFAT signaling in a Parkinson's disease LRRK2 G2019S knock-in model A Wetzel, SH Lei, T Liu, MP Hughes, Y Peng, T McKay, SN Waddington, ... bioRxiv, 2023.03. 31.535090, 2023 | | 2023 |
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 |
CLN7 mutation causes aberrant redistribution of protein isoforms and contributes to Batten disease pathobiology AM Sharaireh, M Guevara-Ferrer, S Herranz-Martin, M Garcia-Macia, ... bioRxiv, 2022.04. 21.488782, 2022 | | 2022 |
Author Correction: Atomic Structures of Segments from TDP-43 LCD and insight into Reversible and Pathogenic Aggregation EL Guenther, Q Cao, H Trinh, J Lu, MR Sawaya, D Cascio, DR Boyer, ... Nature structural & molecular biology 26 (10), 988, 2019 | | 2019 |
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 |