Design principles for bifunctional targeted oligonucleotide enhancers of splicing.
Nucleic Acids Res. 2011 Sep 1; 39(16):7194-208
Judith Sleeman
15 May 2012
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Technical Advance
The modification of splicing of pre-mRNA transcripts is a goal for gene therapy for diseases resulting from aberrant splicing. In the case of spinal muscular atrophy (SMA), the goal is to include exon 7, which is skipped in most transcripts from the SMN2 gene. This article presents a thorough analysis of the efficacy of bifunctional...
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