Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease
Osellame, Laura D., Rahim, Ahad A., Hargreaves, Iain P., Gegg, Matthew E., Richard-Londt, Angela, Brandner, Sebastian, Waddington, Simon N., Schapira, Anthony H.V., Duchen, Michael R.
Published in Cell metabolism (04.06.2013)
Published in Cell metabolism (04.06.2013)
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Journal Article
Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases
Ng, Joanne, Barral, Serena, Waddington, Simon N., Kurian, Manju A.
Published in Movement disorders (01.06.2023)
Published in Movement disorders (01.06.2023)
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Journal Article
NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming
Hawkins, Kate E., Joy, Shona, Delhove, Juliette M.K.M., Kotiadis, Vassilios N., Fernandez, Emilio, Fitzpatrick, Lorna M., Whiteford, James R., King, Peter J., Bolanos, Juan P., Duchen, Michael R., Waddington, Simon N., McKay, Tristan R.
Published in Cell reports (Cambridge) (01.03.2016)
Published in Cell reports (Cambridge) (01.03.2016)
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Journal Article
Gene therapy for neurotransmitter‐related disorders
Chu, Wing Sum, Ng, Joanne, Waddington, Simon N., Kurian, Manju A.
Published in Journal of inherited metabolic disease (01.01.2024)
Published in Journal of inherited metabolic disease (01.01.2024)
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Journal Article
Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice
Suff, Natalie, Karda, Rajvinder, Diaz, Juan A., Ng, Joanne, Baruteau, Julien, Perocheau, Dany, Tangney, Mark, Taylor, Peter W., Peebles, Donald, Buckley, Suzanne M.K., Waddington, Simon N.
Published in The American journal of pathology (01.10.2018)
Published in The American journal of pathology (01.10.2018)
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Journal Article
Fetal gene therapy for neurodegenerative lysosomal storage diseases
Baruteau, Julien, Waddington, Simon N.
Published in Journal of inherited metabolic disease (01.05.2019)
Published in Journal of inherited metabolic disease (01.05.2019)
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Journal Article
Activator protein 1 is a key terminal mediator of inflammation-induced preterm labor in mice
MacIntyre, David A, Lee, Yun S, Migale, Roberta, Herbert, Bronwen R, Waddington, Simon N, Peebles, Donald, Hagberg, Henrik, Johnson, Mark R, Bennett, Phillip R
Published in The FASEB journal (01.05.2014)
Published in The FASEB journal (01.05.2014)
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Journal Article
Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model
Wetzel, Andrea, Lei, Si Hang, Liu, Tiansheng, Hughes, Michael P., Peng, Yunan, McKay, Tristan, Waddington, Simon N., Grannò, Simone, Rahim, Ahad A., Harvey, Kirsten
Published in Scientific reports (29.05.2024)
Published in Scientific reports (29.05.2024)
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Journal Article
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease
Massaro, Giulia, Geard, Amy F, Nelvagal, Hemanth R, Gore, Katrina, Clemo, Nadine K, Waddington, Simon N, Rahim, Ahad A
Published in Human molecular genetics (16.05.2024)
Published in Human molecular genetics (16.05.2024)
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Journal Article
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
Tordo, Julie, O'Leary, Claire, Antunes, André S L M, Palomar, Nuria, Aldrin-Kirk, Patrick, Basche, Mark, Bennett, Antonette, D'Souza, Zelpha, Gleitz, Hélène, Godwin, Annie, Holley, Rebecca J, Parker, Helen, Liao, Ai Yin, Rouse, Paul, Youshani, Amir Saam, Dridi, Larbi, Martins, Carla, Levade, Thierry, Stacey, Kevin B, Davis, Daniel M, Dyer, Adam, Clément, Nathalie, Björklund, Tomas, Ali, Robin R, Agbandje-McKenna, Mavis, Rahim, Ahad A, Pshezhetsky, Alexey, Waddington, Simon N, Linden, R Michael, Bigger, Brian W, Henckaerts, Els
Published in Brain (London, England : 1878) (01.07.2018)
Published in Brain (London, England : 1878) (01.07.2018)
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Journal Article
Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors
Whiteley, Zoe, Massaro, Giulia, Gkogkos, Georgios, Gavriilidis, Asterios, Waddington, Simon N, Rahim, Ahad A, Craig, Duncan Q. M
Published in Nanoscale (23.03.2023)
Published in Nanoscale (23.03.2023)
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Journal Article
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
Perocheau, Dany P, Cunningham, Sharon, Lee, Juhee, Antinao Diaz, Juan, Waddington, Simon N, Gilmour, Kimberly, Eaglestone, Simon, Lisowski, Leszek, Thrasher, Adrian J, Alexander, Ian E, Gissen, Paul, Baruteau, Julien
Published in Human gene therapy (01.01.2019)
Published in Human gene therapy (01.01.2019)
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Journal Article
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease
Hughes, Michael P, Smith, Dave A, Morris, Lauren, Fletcher, Claire, Colaco, Alexandria, Huebecker, Mylene, Tordo, Julie, Palomar, Nuria, Massaro, Giulia, Henckaerts, Els, Waddington, Simon N, Platt, Frances M, Rahim, Ahad A
Published in Human molecular genetics (01.09.2018)
Published in Human molecular genetics (01.09.2018)
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Journal Article
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
Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A
Published in Human molecular genetics (29.07.2020)
Published in Human molecular genetics (29.07.2020)
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Journal Article
Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology
Suleman, Saqlain, Schrubaji, Kuteiba, Filippou, Chrysovalanto, Ignatova, Svetlana, Hewitson, Peter, Huddleston, Jonathan, Karda, Rajvinder, Waddington, Simon N., Themis, Michael
Published in Journal of virological methods (01.01.2022)
Published in Journal of virological methods (01.01.2022)
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Journal Article
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
McIntosh, Jenny, Lenting, Peter J., Rosales, Cecilia, Lee, Doyoung, Rabbanian, Samira, Raj, Deepak, Patel, Nishil, Tuddenham, Edward G.D., Christophe, Olivier D., McVey, John H., Waddington, Simon, Nienhuis, Arthur W., Gray, John T., Fagone, Paolo, Mingozzi, Federico, Zhou, Shang-Zhen, High, Katherine A., Cancio, Maria, Ng, Catherine Y.C., Zhou, Junfang, Morton, Christopher L., Davidoff, Andrew M., Nathwani, Amit C.
Published in Blood (25.04.2013)
Published in Blood (25.04.2013)
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Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload
Plotegher, Nicoletta, Perocheau, Dany, Ferrazza, Ruggero, Massaro, Giulia, Bhosale, Gauri, Zambon, Federico, Rahim, Ahad A., Guella, Graziano, Waddington, Simon N., Szabadkai, Gyorgy, Duchen, Michael R.
Published in Cell death and differentiation (01.05.2020)
Published in Cell death and differentiation (01.05.2020)
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Journal Article
In Utero Gene Therapy Consensus Statement from the IFeTIS
Almeida-Porada, Graça, Waddington, Simon N., Chan, Jerry K.Y., Peranteau, William H., MacKenzie, Tippi, Porada, Christopher D.
Published in Molecular therapy (10.04.2019)
Published in Molecular therapy (10.04.2019)
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