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

Bằng Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul
Xuất bản năm J Inherit Metab Dis (2017)

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

Bằng Waddington, Simon N., Privolizzi, Riccardo, Karda, Rajvinder, O’Neill, Helen C.
Xuất bản năm Curr Stem Cell Rep (2016)

Desmin-regulated Lentiviral Vectors for Skeletal Muscle Gene Transfer

Bằng Talbot, Gillian E, Waddington, Simon N, Bales, Olivia, Tchen, Rose C, Antoniou, Michael N
Được phát hành 2009

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

Bằng Ahmed, Sherif G., Waddington, Simon N., Boza-Morán, Maria Gabriela, Yáñez-Muñoz, Rafael J.
Xuất bản năm J Control Release (2018)

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

Bằng Al-Allaf, Faisal A, Coutelle, Charles, Waddington, Simon N, David, Anna L, Harbottle, Richard, Themis, Michael
Được phát hành 2010

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

Bằng Karda, Rajvinder, Counsell, John R., Karbowniczek, Kinga, Caproni, Lisa J., Tite, John P., Waddington, Simon N.
Xuất bản năm Gene Ther (2019)

Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo

Bằng Disterer, Petra, Al-Shawi, Raya, Ellmerich, Stephan, Waddington, Simon N, Owen, James S, Simons, J Paul, Khoo, Bernard
Được phát hành 2013

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

Bằng Pirianov, Grisha, MacIntyre, David A, Lee, Yun, Waddington, Simon N, Terzidou, Vasso, Mehmet, Huseyin, Bennett, Phillip R
Xuất bản năm Reproduction (2015)

In Utero Gene Therapy Consensus Statement from the IFeTIS

Bằng Almeida-Porada, Graça, Waddington, Simon N., Chan, Jerry K.Y., Peranteau, William H., MacKenzie, Tippi, Porada, Christopher D.
Xuất bản năm Mol Ther (2019)

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

Bằng Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Xuất bản năm Biomolecules (2021)

Targeting of Adenovirus Serotype 5 (Ad5) and 5/47 Pseudotyped Vectors In Vivo: Fundamental Involvement of Coagulation Factors and Redundancy of CAR Binding by Ad5

Bằng Waddington, Simon N., Parker, Alan L., Havenga, Menzo, Nicklin, Stuart A., Buckley, Suzanne M. K., McVey, John H., Baker, Andrew H.
Được phát hành 2007

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

Bằng Baruteau, Julien, Khalil, Youssef, Grunewald, Stephanie, Zancolli, Marta, Chakrapani, Anupam, Cleary, Maureen, Davison, James, Footitt, Emma, Waddington, Simon N., Gissen, Paul, Mills, Philippa
Xuất bản năm Metabolites (2019)

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

Bằng Santos, Chloe, Pai, Yun Jin, Mahmood, M. Raasib, Leung, Kit-Yi, Savery, Dawn, Waddington, Simon N., Copp, Andrew J., Greene, Nicholas D.E.
Xuất bản năm J Clin Invest (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

Bằng Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A
Xuất bản năm Hum Mol Genet (2020)

Perinatal Gene Transfer to the Liver

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

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

Bằng Nivsarkar, Megha S., Buckley, Suzanne M. K., Parker, Alan L., Perocheau, Dany, McKay, Tristan R., Rahim, Ahad A., Howe, Steven J., Waddington, Simon N.
Xuất bản năm J Immunol Res (2015)

Gene Delivery of a Mutant TGFβ3 Reduces Markers of Scar Tissue Formation After Cutaneous Wounding

Bằng Waddington, Simon N, Crossley, Rachel, Sheard, Vicky, Howe, Steven J, Buckley, Suzanne MK, Coughlan, Lynda, Gilham, David E, Hawkins, Robert E, McKay, Tristan R
Được phát hành 2010

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

Bằng 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.
Xuất bản năm Cell Death Differ (2019)

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

Bằng 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.
Xuất bản năm Cell Death Differ (2020)

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

Bằng 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.
Xuất bản năm Mol Ther Methods Clin Dev (2020)