Microsphere-Based Therapy for Reversing New-Onset Autoimmune Diabetes
This invention is an innovative microsphere-based composition that delivers antisense oligonucleotides to modulate the immune system. It can reverse autoimmune insulin-dependent diabetes by suppressing the expression of key T-cell costimulatory molecules.
Description
The technology utilizes a microsphere-based delivery system to administer antisense oligonucleotides. These microspheres, fabricated using a specialized aqueous process, contain specific antisense oligonucleotides that target primary transcripts for CD40, CD80, and CD86. Upon injection, the microspheres are actively taken up by dendritic cells. The antisense oligonucleotides then suppress the expression of the CD40, CD80, and CD86 cell surface molecules in these dendritic cells. This down-regulation of costimulatory molecules effectively modulates dendritic cell activity and mobilizes regulatory networks. The in vivo administration of these microspheres has been shown to reverse the disease in a relevant animal model.Applications
- Treatment for autoimmune diabetes (e.g., Type 1 diabetes).- Therapeutic for conditions that require targeted immune modulation through the suppression of CD40, CD80, and CD86.
- Advanced drug delivery system for nucleic acid therapies.
Advantages
- Disease Reversal: This therapy can normalize blood glucose levels and reverse new-onset autoimmune diabetes in a non-human animal model.- Targeted Immune Modulation: The microspheres specifically down-regulate key costimulatory molecules, modulating the immune system's activity without causing severe adverse immune responses.
- Improved Delivery: The composition addresses the challenges of delivering nucleic acids in vivo by protecting them from degradation and exhibiting low toxicity compared to other methods.
- Convenient Administration: The microspheres are designed for a syringe based delivery route, allowing for injection through fine bore needles.