Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy

by Goyenvalle, Aurélie, Wright, Jordan, Babbs, Arran, Wilkins, Vivienne, Garcia, Luis, Davies, Kay E
Published 2012

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery

by Eckenfelder, Agathe, Tordo, Julie, Babbs, Arran, Davies, Kay E, Goyenvalle, Aurélie, Danos, Olivier
Published 2012

Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD Therapy

by Goyenvalle, Aurélie, Babbs, Arran, van Ommen, Gert-Jan B, Garcia, Luis, Davies, Kay E
Published 2009

Temporal transcriptomics suggest that twin-peaking genes reset the clock

by Pembroke, William G, Babbs, Arran, Davies, Kay E, Ponting, Chris P, Oliver, Peter L
Published in eLife (2015)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

by Goyenvalle, Aurélie, Babbs, Arran, Wright, Jordan, Wilkins, Vivienne, Powell, Dave, Garcia, Luis, Davies, Kay E.
Published 2012

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

by Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E
Published 2009

Identification of serum protein biomarkers for utrophin based DMD therapy

by Guiraud, Simon, Edwards, Benjamin, Squire, Sarah E., Babbs, Arran, Shah, Nandini, Berg, Adam, Chen, Huijia, Davies, Kay E.
Published in Sci Rep (2017)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy

by Guiraud, Simon, Edwards, Benjamin, Babbs, Arran, Squire, Sarah E, Berg, Adam, Moir, Lee, Wood, Matthew J, Davies, Kay E
Published in Hum Mol Genet (2019)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD

by Guiraud, Simon, Edwards, Benjamin, Squire, Sarah E, Moir, Lee, Berg, Adam, Babbs, Arran, Ramadan, Nesrine, Wood, Matthew J, Davies, Kay E
Published in Hum Mol Genet (2019)

From diagnosis to therapy in Duchenne muscular dystrophy

by Babbs, Arran, Chatzopoulou, Maria, Edwards, Ben, Squire, Sarah E., Wilkinson, Isabel V.L., Wynne, Graham M., Russell, Angela J., Davies, Kay E.
Published in Biochem Soc Trans (2020)

Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice

by Kennedy, Tahnee L., Guiraud, Simon, Edwards, Ben, Squire, Sarah, Moir, Lee, Babbs, Arran, Odom, Guy, Golebiowski, Diane, Schneider, Joel, Chamberlain, Jeffrey S., Davies, Kay E.
Published in Mol Ther Methods Clin Dev (2018)

A dominant mutation in Snap25 causes impaired vesicle trafficking, sensorimotor gating, and ataxia in the blind-drunk mouse

by Jeans, Alexander F., Oliver, Peter L., Johnson, Reuben, Capogna, Marco, Vikman, Jenny, Molnár, Zoltán, Babbs, Arran, Partridge, Christopher J., Salehi, Albert, Bengtsson, Martin, Eliasson, Lena, Rorsman, Patrik, Davies, Kay E.
Published 2007

Second-generation compound for the modulation of utrophin in the therapy of DMD

by Guiraud, Simon, Squire, Sarah E., Edwards, Benjamin, Chen, Huijia, Burns, David T., Shah, Nandini, Babbs, Arran, Davies, Stephen G., Wynne, Graham M., Russell, Angela J., Elsey, David, Wilson, Francis X., Tinsley, Jon M., Davies, Kay E.
Published in Hum Mol Genet (2015)

Synthesis of SMT022357 enantiomers and in vivo evaluation in a Duchenne muscular dystrophy mouse model

by Babbs, Arran, Berg, Adam, Chatzopoulou, Maria, Davies, Kay E., Davies, Stephen G., Edwards, Benjamin, Elsey, David J., Emer, Enrico, Figuccia, Aude L.A., Fletcher, Ai M., Guiraud, Simon, Harriman, Shawn, Moir, Lee, Robinson, Neil, Rowley, Jessica A., Russell, Angela J., Squire, Sarah E., Thomson, James E., Tinsley, Jonathon M., Wilson, Francis X., Wynne, Graham M.
Published in Tetrahedron (2020)