Part of the Progress in Molecular and Subcellular Biology book series (PMSB, volume 44)
Alternative Splicing: Therapeutic Target and Tool
KeywordsAlternative Splice Cystic Fibrosis Transmembrane Conductance Regulator Duchenne Muscular Dystrophy Spinal Muscular Atrophy Survival Motor Neuron
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.
Unable to display preview. Download preview PDF.
- De Angelis FG, Sthandier O et al. (2002) Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells. Proc Natl Acad Sci USA 99(14): 9456-9461PubMedGoogle Scholar
- Eperon IC, Muntoni F (2003) Response to Buratti et al.: Can a ‘patch’ in a skipped exon make the pre-mRNA splicing machine run better? Trends Mol Med 9 (6): 233-234Google Scholar
- Garcia Blanco M (2005) Making antisense of splicing. Current Opinion in Molecular Therapeutics, in pressGoogle Scholar
- Vitarene Study Group (2002) A randomized controlled clinical trial of intravitre-ous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS. Am J Ophthalmol 133(4): 467-474Google Scholar
© Springer Berlin Heidelberg 2006