Therapeutic splice modification

Jon Moulton

Thursday, 08 Jan 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).


Search forums Advanced search

web feed

Submit this topic to

Advertisement