原创力文档assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy外显子的可行性评估45 - 55 multiexon跳过杜氏肌肉营养不良症.pdfVIP
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assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy外显子的可行性评估45 - 55 multiexon跳过杜氏肌肉营养不良症
BMC Medical Genetics BioMed Central
Research article Open Access
Assessment of the feasibility of exon 45–55 multiexon skipping for
duchenne muscular dystrophy
1 1 2
Laura van Vliet , Christa L de Winter , Judith CT van Deutekom , Gert-
1 1
Jan B van Ommen and Annemieke Aartsma-Rus*
Address: 1DMD Genetic Therapy Group, Department of Human Genetics, Leiden University, Medical Center, Postzone S4-P, PO Box 9600,
2300RC, Leiden, The Netherlands and 2Prosensa Therapeutics B.V, Wassenaarseweg 72, 2333AL, Leiden, The Netherlands
Email: Laura van Vliet - l.van_vliet@lumc.nl; Christa L de Winter - c.l.de_winter@lumc.nl; Judith CT van
Deutekom - j.vandeutekom@prosensa.nl; Gert-Jan B van Ommen - gjvo@lumc.nl; Annemieke Aartsma-Rus* - a.m.rus@lumc.nl
* Corresponding author
Published: 1 December 2008 Received: 20 February 2008
Accepted: 1 December 2008
BMC Medical Genetics 2008, 9:105 doi:10.1186/1471-2350-9-105
This article is available from: /1471-2350/9/105
© 2008 van Vliet et al; licensee BioMed Central Ltd.
This is an Open Access article distributed under the terms of the Creative Commons Attribution License (/licenses/by/2.0),
which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Abstract
Background: The specific skipping of an exon, induced by antisense oligonucleotides (AON)
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