Therapeutic splice modification
Jon Moulton
Thursday, 08 January 2009 20:53 UTC
Bauman J, Jearawiriyapaisarn N, Kole R. Therapeutic Potential of Splice-Switching Oligonucleotides. Oligonucleotides. 2009 Jan 6. [Epub ahead of print]
http://www.ncbi.nlm.nih.gov/pubmed/19125639
Ryszard Kole of AVI BioPharma Inc. has published this review of therapeutic applications of splice-modifying oligos, including information about Morpholinos targeting Duchenne muscular dystrophy.
This review also explores a number of other potential therapeutic applications of splice-modifying Morpholinos, including discussions of targets such as β-globin (β-Thalassemia), SMN2 (Spinal muscular atrophy), TNFR2 and MyD88 (Inflammatory diseases), HER2 (Cancer), ClC-1 (Dystrophia myotonica type 1), ATP7A (Menkes disease), ATM (Ataxia telangiectasia), APOB (Atherosclerosis), PCCA, PCCB, and MUT (Propionic and methylmalonic acidemias).
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