Personalised genetic medicines for inherited disorders

• In: SYMPOSIUM: GENE-BASED THERAPEUTICS: POSSIBILITIES AND CHALLENGES on Monday, 14 April 2014, 13:00-14:30
• At: PSWC, Melbourne (Australia) (2014)
• 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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