Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
- PMID: 23188050
- PMCID: PMC3722956
- DOI: 10.1038/ejhg.2012.264
Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
Comment on
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In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.Eur J Hum Genet. 2013 Jul;21(7):769-73. doi: 10.1038/ejhg.2012.261. Epub 2012 Nov 28. Eur J Hum Genet. 2013. PMID: 23188051 Free PMC article.
References
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- Anthony K, Feng L, Arechavala-Gomeza V, et al. Exon skipping quantification by qRT-PCR in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen Hum Gene Ther Methods 2012(e-pub ahead of print). - PubMed
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- Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011;364:1513–1522. - PubMed
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