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Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
Published on: May 24, 2016
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Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping
Dunhui Li1, Frank L Mastaglia2, Sue Fletcher1
1Centre for Comparative Genomics, Murdoch University, Perth 6050, Australia; Perron Institute for Neurological and Translational Science, University of Western Australia, Perth 6000, Australia.
Trends in Pharmacological Sciences
|October 5, 2018
Summary
Antisense oligonucleotides (ASOs) show promise for treating rare genetic diseases by skipping specific exons. This approach, exemplified by Duchenne muscular dystrophy (Duchenne MD), offers precision medicine for inherited disorders.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Antisense RNA therapeutics, such as Exondys51® for Duchenne muscular dystrophy (Duchenne MD) and Spinraza® for spinal muscular atrophy (SMA), are now in clinical use.
- These therapies highlight the potential of antisense oligonucleotides (ASOs) in treating genetic disorders.
Purpose of the Study:
- To review the potential of therapeutic alternative splicing, focusing on exon-skipping ASOs.
- To explore the application of exon-skipping ASOs in Duchenne MD and other inherited rare diseases.
Main Methods:
- Review of existing literature on ASO therapeutics and exon skipping.
- Analysis of Duchenne MD as a model for targeted exon-skipping strategies.
- Speculation on novel applications for rare diseases with dispensable exons.
Main Results:
- Exon-skipping ASOs can specifically remove dispensable exons, potentially bypassing harmful mutations.
- This approach can lead to clinical benefits for patients with amenable mutations.
- The strategy holds promise for precision medicine in various inherited rare diseases.
Conclusions:
- Therapeutic alternative splicing, particularly exon skipping via ASOs, is a viable strategy for genetic diseases.
- Duchenne MD serves as a key example, with potential applications extending to other rare inherited conditions.
- Targeted exon skipping represents a significant advancement in precision medicine for rare genetic disorders.
Keywords:
antisense oligonucleotidesdispensable exonsinherited rare diseasesprecision medicinetherapeutic alternative splicingMore Related Videos
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