Nucleic Acid Delivery

Research Area: Novel biomaterials

RNAMEDs is developing clinically relevant delivery system based on novel biomaterials to facilitate effective RNA-based therapies, including degradable polymers, modified cyclodextrins, and lipid-like materials.

Investigators

Key Papers

Prof Caitriona O'Driscoll and Dr Piotr Kowalski 

Ionizable Amino-polyesters synthesized   via ring opening polymerization of tertiary amino-alcohols for a tissue selective mRNA delivery.

Cyclodextrin-siRNA conjugates as versatile gene silencing agents.

PEGylated cyclodextrins as Novel siRNA nanosystems: correlations between polyethylene glycol length and nanoparticle stability.

A click chemistry route to 2-functionalised PEGylated and cationic Beta-cyclodextrins: co-formulation opportunities for siRNA delivery.

Endothelial siRNA delivery in nonhuman primates using ionizable low–molecular weight polymeric nanoparticles

 

 Research Area: New RNA cargo

We are utilising various types of RNA cargos for specific and durable control of protein expression in target cells such as short interfering RNA (siRNA), messenger RNA (mRNA), circularRNA (circRNA)

Investigators

Key Papers

Prof Caitriona O'Driscoll and Dr Piotr Kowalski 

Engineering Circular RNA for Potent and Stable Translation in Eukaryotic Cells.

RNA circularization diminishes immunogenicity and can extend translation duration in vivo.

Self-Assembled Cationic β‑Cyclodextrin Nanostructures for siRNA Delivery.

Folate-targeted amphiphilic cyclodextrin nanoparticles incorporating a fusogenic peptide deliver therapeutic siRNA and inhibit the invasive capacity of 3D prostate cancer tumours.

 

Research Area: Therapeutic areas

We are exploring the breakthrough therapeutic potential of RNA molecules for preventing multiple organ failure during sepsis, treatment of cancer (solid tumours and blood cancers), cardiovascular, neurodegenerative and inflammatory bowel diseases.

Investigator

Key Papers

Prof Caitriona O'Driscoll and Dr Piotr Kowalski 

Optimization of mRNA-Based delivery system for Anti-Her2 antibody expression in vivo

Anti-VCAM-1 SAINT-O-Somes enable endothelial-specific delivery of siRNA and downregulation of inflammatory genes in activated endothelium in vivo

Antibody-targeted cyclodextrin-based nanoparticles for siRNA delivery in the treatment of acute myeloid leukaemia – physicochemical characteristics, in vitro mechanistic studies and ex vivo patient derived therapeutic efficacy.

Self-assembling Modified Beta-Cyclodextrin Nanoparticles as Neuronal siRNA Delivery Vectors: Focus on Huntington's Disease.

Gene silencing of TNF-alpha in a murine model of acute colitis using a modified cyclodextrin delivery system.

 

Research Area: Oral delivery

We are addressing challenges for clinical translation for RNA therapeutic administration via non-invasive routes, such as oral delivery.

Investigator

Key Papers

Prof Caitriona O'Driscoll and Dr Piotr Kowalski 

Oral delivery of non-viral nucleic acid-based therapeutics - do we have the guts for this?

Gastrointestinal gene delivery by Modified Cyclodextrins - in vitro identification and quantification of extracellular barriers.

 

Platform capabilities:

  • Synthesis and characterization of polymeric and lipid-like biomaterials
  • Design, formulation and characterization of non-viral systems for nucleic acids delivery
  • Microfluidic based formulation of nanoparticles
  • Processing; Freeze/Thaw, Lyophilisation
  • Design, synthesis, and characterization of therapeutic RNAs
  • Bio-predictive in-vitro and in-vivo testing of siRNA/mRNA formulations
  • Pharmacological evaluation of the safety and efficacy of RNA therapeutics in disease relevant animal models (e.g.)               

School of Pharmacy

Cógaisíocht

Cavanagh Pharmacy Building Room UG06 University College Cork

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