Investigating the use of oligonucleotides for the treatment of muscular dystrophy
Moore, Rebecca L. L. (2016) Investigating the use of oligonucleotides for the treatment of muscular dystrophy. PhD thesis, University of Nottingham.
Antisense oligonucleotide therapy is one of the most promising strategies for treatment of myotonic dystrophy type 1 (DM1), type 2 (DM2) and Duchene muscular dystrophy (DMD). These three diseases have nuclear retained mRNA, suitable for antisense therapy targeting. The delivery of oligonucleotides to their desired target has long been an obstacle in antisense therapy with a large number of delivery reagents or methods having adverse side effects.
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