Multiplicity of experimental approaches to therapy for genetic muscle diseases and necessity for population screening
Author:
Publisher
Springer Science and Business Media LLC
Subject
Cell Biology,Biochemistry,Physiology
Link
http://link.springer.com/content/pdf/10.1007/s10974-008-9158-5.pdf
Reference62 articles.
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2. Arechavala-Gomeza V, Graham IR, Popplewell LJ et al (2007) Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther 18:798–810. doi: 10.1089/hum.2006.061
3. Assereto S, Stringara S, Sotgia F et al (2006) Pharmacological rescue of the dystrophin-glycoprotein complex in Duchenne and Becker skeletal muscle explants by proteasome inhibitor treatment. Am J Physiol Cell Physiol 290:C577–C582. doi: 10.1152/ajpcell.00434.2005
4. Bartoli M, Gicquel E, Barrault L et al (2008) Mannosidase I inhibition rescues the human alpha-sarcoglycan R77C recurrent mutation. Hum Mol Genet 17:1214–1221. doi: 10.1093/hmg/ddn029
5. Beckmann R, Sauer M, Ketelsen U-P et al (1978) Early diagnosis of Duchenne muscular dystrophy. Lancet ii:105. doi: 10.1016/S0140-6736(78)91419-8
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