Chemistry BSc students contributing to curative treatment for rare disease

Last year, ten BSc students from the School of Chemistry (Hasan Abosoglu, Alfeshani Cassim, Edmund Chu, Simon Doughty, Shaun-Jones Eganda, Hikari Fuchigami, Aleah Hussein, Meedhu Olickal, Neelay Parmar and Akhil Sebastian) under the supervision of Prof Sergey Piletsky, Dr Kal Karim, Dr Elena Piletska and PhD student Fabiana Grillo, contributed to the CureCN project, which was funded by the EU’s Horizon2020 research and innovation program (award number: 755225). The main objective of the project was to produce a tool for improving the efficiency of gene therapy used in the treatment of Crigler-Najjar (CN) syndrome. CN syndrome is a genetic disorder characterised by an inability or diminished ability to convert and clear bilirubin from the body.

The aim of the students’ work was to develop synthetic binders for adeno-associated virus (AAV) - neutralising antibodies. These materials are in high demand as a solution to one of the biggest problems associated with the practical application of gene therapy in genetic disorders – the decreasing efficiency of the treatment over time because of the patient’s immune response. A combinatorial library of fluorescent polymeric nanoparticles was prepared and screened against six peptide epitopes of the variable regions of the AAV - neutralising antibodies, which were identified by the Leicester Biotechnology Group, a partner in the EU project. Students provided a thorough investigation of the components of the monomeric mixture that had a positive or negative impact on polymer binding to the peptides with a different composition and properties. This study produced valuable information for experts who use material engineering to solve practical problems related to nanotechnology and drug delivery.

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