Tuesday, 27 March 2012

Seminar: antisense approaches as a therapy for muscular dystrophy

Wednesday 28th March 2012, 13:15 – 14:15
Perrin Lecture Theatre, 4 Newark Street, Whitechapel
Lunch will be provided
University College London  

“Antisense approaches as a therapy for muscular dystrophy”

Professor Muntoni graduated in Medicine in Italy in 1984 and obtained his specialization in Child Neurology and Psychiatry in 1989. Since 1993 he has worked in London, initially as a Lecturer (1993); Senior Lecturer (1994); Reader (1996) and Professor of Paediatric Neurology at the Hammersmith Hospital, Imperial College. In 2007 he moved the clinical, pathology and academic group from Imperial College to UCL Institute of Child Health and Great Ormond Street Hospital for Children. He is director of the Dubowitz Neuromuscular Centre, leading national and international centre for the diagnosis, management, treatment and experimental research therapy development in childhood neuromuscular disorders. The Centre has very close clinical and academic links with the adult neuromuscular centre at the Institute of Neurology, also at UCL.


1) Cirak S et al. Exon skipping and dystrophin restoration in Duchenne Muscular Dystrophy patients after systemic phosphorodiamidate morpholino oligomer treatment. The Lancet, 2011;378:595-605.
2) Cirak S et al .Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy. Mol Ther. 2011 Nov 15
3) Anthony K et al. Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials. Brain. 2011 Dec;134(Pt 12):3544-56.
4) Kinali M et al. Local Restoration of Dystrophin Expression in Duchenne Muscular Dystrophy: A Single Blind, Placebo-controlled Dose Escalation Study Using Morpholino Antisense Oligomer AVI-4658. Lancet Neurol. 2009 8(10):918-28.
Coordinator: Professor Dean Nizetic

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