L. Price

Applications of antisense oligonucleotides in designing a therapy for spinal muscular atrophy

L. Price, 2017 .

Co-regulation of survival of motor neuron and Bcl-xL expression: Implications for neuroprotection in spinal muscular atrophy

F. Mastaglia, S. Boulos, B. Meloni, 2012, Neuroscience.

Translational development of splice-modifying antisense oligomers

M. Bellgard, S. Wilton, S. Fletcher, 2017, Expert opinion on biological therapy.

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

F. Muntoni, Haiyan Zhou, S. Wilton, 2013, PloS one.

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice.

F. Muntoni, I. Eperon, K. Anthony, 2013, Human gene therapy.

Complement-mediated muscle cell lysis: A possible mechanism of myonecrosis in anti-SRP associated necrotizing myopathy (ASANM)

F. Mastaglia, S. Wilton, C. Mitrpant, 2013, Journal of Neuroimmunology.