Saturday 19 July 2014

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript

Splice-switching targeted oligonucleotide drugs alter editing of gene transcript: "In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already led to promising treatments for such diseases as Duchenne Muscular Dystrophy and spinal muscular atrophy, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website."



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