- January 10, 2023
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Time: 8AM Pacific | 11AM Eastern | 5PM CET
Date: May 18, 2023
Remko will outline our findings concerning the relationship between the splicing speed of DMD introns and the ability of AONs to induce exon skipping. In the past we have described that the introns of the DMD gene, which is the largest gene in the human genome and is causative for Duchenne muscular dystrophy when mutated, are not spliced sequentially. We used an extensive library of PMO AONs targeting different DMD exons to test whether this splicing speed is correlated with AON-mediated exon skipping efficiency.
Cell-specific targeting and cytoplasmic delivery of small interfering RNA (siRNA) to non-hepatic tissues remain major obstacles to the clinical application of RNA interference. Cationic lipid carrier delivery methods are inefficient, lack cell specificity, and are associated with toxic effects on the cells of interest. SRI Biosciences has developed the Fox Three Platform to move beyond these obstacles to siRNA delivery. Using this platform, we have identified novel peptidic molecular guidance systems (MGSs) to enable cell-specific delivery of biotherapeutic payloads including siRNA. The chemically optimized MGSs are simple to manufacture and can accommodate modifications for conjugation to specific therapeutic payloads. This brief seminar highlights the cell-specific delivery of effective siRNA payloads to distinct tumor cell types with resulting knockdown (KD) of the targeted gene.
- Conjugation of the siRNA payload to the MGS peptide does not alter the cell-specific uptake of the
- MGS-siRNA conjugates suppress targeted gene expression with no need for a lipid carrier.
- Our work demonstrates effective cell-specific delivery of functional siRNA.