期刊
EMBO MOLECULAR MEDICINE
卷 9, 期 5, 页码 545-557出版社
WILEY
DOI: 10.15252/emmm.201607199
关键词
antisense oligonucleotides; delivery; pre-clinical models; RNA therapy; toxicity
资金
- Sarepta Therapeutics, US
- British Heart Foundation [PG/12/20/29469] Funding Source: researchfish
- Medical Research Council [G0802469] Funding Source: researchfish
- MRC [G0802469] Funding Source: UKRI
The use of splice-switching antisense therapy is highly promising, with a wealth of pre-clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The main obstacle impeding the clinical translation of this approach is the relatively poor delivery of antisense oligonucleotides to target tissues after systemic delivery. We are a group of researchers closely involved in the development of these therapies and would like to communicate our discussions concerning the validity of standard methodologies currently used in their pre-clinical development, the gaps in current knowledge and the pertinent challenges facing the field. We therefore make recommendations in order to focus future research efforts and facilitate a wider application of therapeutic antisense oligonucleotides.
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