University of London

Other

Benitec Biopharma Limited

Teesside University

John George Dickson

Linda Popplewell

Ian Graham

David A Suhy

Michael Graham

Capucine Trollet

Pavlos Alifragis

Alberto Malerba

Amninder Sangha

Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.

Oligomers

The invention relates to neurodegenerative disorders, and in particular to novel oligonucleotides for treating such conditions, for example Alzheimer's disease. The invention provides novel antisense oligonucleotides, and compositions comprising such oligos, and therapies and methods for treating neurodegenerative disorders. The invention includes genome editing techniques for achieving similar results as using the novel antisense oligonucleotides.

Neurodegenerative disorders

The present disclosure relates to RNA interference (RNAi) reagents for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.

Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

Growing community of inventors

Surrey, United Kingdom

John George Dickson