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Personalised genetic medicines for inherited disorders

  • At: 2014 FIP Congress in PSWC, Melbourne (Australia)
  • Type: Presentation
  • By: WILTON, Steve (Murdoch University, Centre for Comparative Genomics, Perth, Australia)
  • Abstract:

    Background. Antisense oligonucleotides can redirect the pre-mRNA processing of targeted gene transcripts. Therapeutic alternative splicing can be employed excise a selected exon or enhance recognition of an exon normally excluded from the mature mRNA.
    Aims. Duchenne muscular dystrophy (DMD), the most common and serious form of childhood muscle..

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Last update 4 October 2019

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