Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches

por Ng, Joanne, Barral, Serena, Waddington, Simon N., Kurian, Manju A.
Publicado 2023

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

por Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul
Publicado 2017

A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells

por Waddington, Simon N., Privolizzi, Riccardo, Karda, Rajvinder, O’Neill, Helen C.
Publicado 2016

High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

por Ahmed, Sherif G., Waddington, Simon N., Boza-Morán, Maria Gabriela, Yáñez-Muñoz, Rafael J.
Publicado 2018

LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives

por Al-Allaf, Faisal A, Coutelle, Charles, Waddington, Simon N, David, Anna L, Harbottle, Richard, Themis, Michael
Publicado 2010

Production of lentiviral vectors using novel, enzymatically produced, linear DNA

por Karda, Rajvinder, Counsell, John R., Karbowniczek, Kinga, Caproni, Lisa J., Tite, John P., Waddington, Simon N.
Publicado 2019

Specific inhibition of c-Jun N-terminal kinase delays preterm labour and reduces mortality

por Pirianov, Grisha, MacIntyre, David A, Lee, Yun, Waddington, Simon N, Terzidou, Vasso, Mehmet, Huseyin, Bennett, Phillip R
Publicado 2015

Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology

por Suleman, Saqlain, Schrubaji, Kuteiba, Filippou, Chrysovalanto, Ignatova, Svetlana, Hewitson, Peter, Huddleston, Jonathan, Karda, Rajvinder, Waddington, Simon N., Themis, Michael
Publicado 2022

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

por Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Publicado 2021

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment

por Baruteau, Julien, Khalil, Youssef, Grunewald, Stephanie, Zancolli, Marta, Chakrapani, Anupam, Cleary, Maureen, Davison, James, Footitt, Emma, Waddington, Simon N., Gissen, Paul, Mills, Philippa
Publicado 2019

Impaired folate 1-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase–deficient mice

por Santos, Chloe, Pai, Yun Jin, Mahmood, M. Raasib, Leung, Kit-Yi, Savery, Dawn, Waddington, Simon N., Copp, Andrew J., Greene, Nicholas D.E.
Publicado 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

por Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A
Publicado 2020

Perinatal Gene Transfer to the Liver

por McKay, Tristan R, Rahim, Ahad A, Buckley, Suzanne M.K, Ward, Natalie J, Chan, Jerry K.Y, Howe, Steven J, Waddington, Simon N
Publicado 2011

Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

por Nivsarkar, Megha S., Buckley, Suzanne M. K., Parker, Alan L., Perocheau, Dany, McKay, Tristan R., Rahim, Ahad A., Howe, Steven J., Waddington, Simon N.
Publicado 2015

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

por 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.
Publicado 2019

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

por 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.
Publicado 2020

Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation

por Counsell, John R., De Brabandere, Guillaume, Karda, Rajvinder, Moore, Marc, Greco, Antonio, Bray, Alysha, Diaz, Juan Antinao, Perocheau, Dany P., Mock, Ulrike, Waddington, Simon N.
Publicado 2020

Regionally-Specified Second Trimester Fetal Neural Stem Cells Reveals Differential Neurogenic Programming

por Fan, Yiping, Marcy, Guillaume, Lee, Eddy S. M., Rozen, Steve, Mattar, Citra N. Z., Waddington, Simon N., Goh, Eyleen L. K., Choolani, Mahesh, Chan, Jerry K. Y.
Publicado 2014

Modeling hormonal and inflammatory contributions to preterm and term labor using uterine temporal transcriptomics

por Migale, Roberta, MacIntyre, David A., Cacciatore, Stefano, Lee, Yun S., Hagberg, Henrik, Herbert, Bronwen R., Johnson, Mark R., Peebles, Donald, Waddington, Simon N., Bennett, Phillip R.
Publicado 2016

Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice

por Delhove, Juliette M. K. M., Buckley, Suzanne M. K., Perocheau, Dany P., Karda, Rajvinder, Arbuthnot, Patrick, Henderson, Neil C., Waddington, Simon N., McKay, Tristan R.
Publicado 2017