Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease von 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.

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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects von Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul

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Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases von Ng, Joanne, Barral, Serena, Waddington, Simon N., Kurian, Manju A.

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NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming von 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.

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The power of bioluminescence imaging in understanding host-pathogen interactions von Suff, Natalie, Waddington, Simon N.

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Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice von 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.

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Gene therapy for neurotransmitter‐related disorders von Chu, Wing Sum, Ng, Joanne, Waddington, Simon N., Kurian, Manju A.

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Activator protein 1 is a key terminal mediator of inflammation‐induced preterm labor in mice von MacIntyre, David A., Lee, Yun S., Migale, Roberta, Herbert, Bronwen R., Waddington, Simon N., Peebles, Donald, Hagberg, Henrik, Johnson, Mark R., Bennett, Phillip R.

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Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model von Wetzel, Andrea, Lei, Si Hang, Liu, Tiansheng, Hughes, Michael P., Peng, Yunan, McKay, Tristan, Waddington, Simon N., Grannò, Simone, Rahim, Ahad A., Harvey, Kirsten

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Fetal gene therapy for neurodegenerative lysosomal storage diseases von Baruteau, Julien, Waddington, Simon N.

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Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease von Massaro, Giulia, Geard, Amy F, Nelvagal, Hemanth R, Gore, Katrina, Clemo, Nadine K, Waddington, Simon N, Rahim, Ahad A

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Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort von 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

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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency von 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

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Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors von Whiteley, Zoe, Massaro, Giulia, Gkogkos, Georgios, Gavriilidis, Asterios, Waddington, Simon N, Rahim, Ahad A, Craig, Duncan Q. M

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AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease von 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

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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 von Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A

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Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer von Baruteau, Julien, Perocheau, Dany P., Hanley, Joanna, Lorvellec, Maëlle, Rocha-Ferreira, Eridan, Karda, Rajvinder, Ng, Joanne, Suff, Natalie, Diaz, Juan Antinao, Rahim, Ahad A., Hughes, Michael P., Banushi, Blerida, Prunty, Helen, Hristova, Mariya, Ridout, Deborah A., Virasami, Alex, Heales, Simon, Howe, Stewen J., Buckley, Suzanne M. K., Mills, Philippa B., Gissen, Paul, Waddington, Simon N.

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Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease von Pauzuolyte, Valda, Patel, Aara, Wawrzynski, James R, Ingham, Neil J, Leong, Yeh Chwan, Karda, Rajvinder, Bitner‐Glindzicz, Maria, Berger, Wolfgang, Waddington, Simon N, Steel, Karen P, Sowden, Jane C

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Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches von Ng, Joanne, Barral, Serena, Waddington, Simon N, Kurian, Manju A

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Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders von Soria, Leandro R, Gurung, Sonam, De Sabbata, Giulia, Perocheau, Dany P, De Angelis, Angela, Bruno, Gemma, Polishchuk, Elena, Paris, Debora, Cuomo, Paola, Motta, Andrea, Orford, Michael, Khalil, Youssef, Eaton, Simon, Mills, Philippa B, Waddington, Simon N, Settembre, Carmine, Muro, Andrés F, Baruteau, Julien, Brunetti‐Pierri, Nicola

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