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)
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.. The access to the whole abstract and if available the presentation file is available to FIP members and to congress participants of that specific congress.