- Glenn Crusea,b,1,
- Yuzhi Yinb,
- Tomoki Fukuyamaa,
- Avanti Desaib,
- Greer K. Arthura,
- Wolfgang Bäumera,
- Michael A. Beavenc, and
- Dean D. Metcalfeb
- Edited by K. Frank Austen, Brigham and Women's Hospital, Boston, MA, and approved October 26, 2016 (received for review May 27, 2016)
Significance
We identified an innovative use for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells. Exon skipping is typically used as part of personalized medicine, where a mutant exon is skipped after sequencing the patients’ affected genes. Our approach, however, targets a nonmutated gene and an exon that is critical for surface IgE receptor expression. It does not require a personalized approach with genetic sequencing or multiple iterations of oligonucleotides that would require clinical trials. Furthermore, the diseases to be treated with this technology are ideal for local delivery of the oligonucleotides by aerosols or topical cream formulations.