Journal article
Translational development of splice-modifying antisense oligomers
Expert Opinion on Biological Therapy, Vol.17(1), pp.15-30
2017
Abstract
Introduction: Antisense nucleic acid analogues can interact with pre-mRNA motifs and influence exon or splice site selection and thereby alter gene expression. Design of antisense molecules to target specific motifs can result in either exon exclusion or exon inclusion during splicing. Novel drugs exploiting the antisense concept are targeting rare, life-limiting diseases; however, the potential exists to treat a wide range of conditions by antisense-mediated splice intervention.
Areas covered: In this review, the authors discuss the clinical translation of novel molecular therapeutics to address the fatal neuromuscular disorders Duchenne muscular dystrophy and spinal muscular atrophy. The review also highlights difficulties posed by issues pertaining to restricted participant numbers, variable phenotype and disease progression, and the identification and validation of study endpoints.
Expert opinion: Translation of novel therapeutics for Duchenne muscular dystrophy and spinal muscular atrophy has been greatly advanced by multidisciplinary research, academic-industry partnerships and in particular, the engagement and support of the patient community. Sponsors, supporters and regulators are cooperating to deliver new drugs and identify and define meaningful outcome measures. Non-conventional and adaptive trial design could be particularly suited to clinical evaluation of novel therapeutics and strategies to treat serious, rare diseases that may be problematic to study using more conventional clinical trial structures.
Details
- Title
- Translational development of splice-modifying antisense oligomers
- Authors/Creators
- S. Fletcher (Author/Creator) - The University of Western AustraliaM.I. Bellgard (Author/Creator) - Perron Institute for Neurological and Translational ScienceL. Price (Author/Creator) - The University of Western AustraliaA.P. Akkari (Author/Creator) - Western Australian Neuroscience Research Institute, Nedlands, Western Australia, Australia; Centre for Comparative Genomics, Murdoch University, Western Australia, Australia; Shiraz Pharmaceuticals, Inc, Chapel Hill, NC, USAS.D. Wilton (Author/Creator) - The University of Western Australia
- Publication Details
- Expert Opinion on Biological Therapy, Vol.17(1), pp.15-30
- Publisher
- Taylor & Francis
- Identifiers
- 991005544368207891
- Murdoch Affiliation
- Centre for Comparative Genomics
- Language
- English
- Resource Type
- Journal article
UN Sustainable Development Goals (SDGs)
This output has contributed to the advancement of the following goals:
Source: InCites
Metrics
20 Record Views
InCites Highlights
These are selected metrics from InCites Benchmarking & Analytics tool, related to this output
- Collaboration types
- Domestic collaboration
- International collaboration
- Citation topics
- 1 Clinical & Life Sciences
- 1.255 Musculoskeletal Disorders
- 1.255.628 Duchenne Muscular Dystrophy
- Web Of Science research areas
- Biotechnology & Applied Microbiology
- Medicine, Research & Experimental
- ESI research areas
- Clinical Medicine