Biodiscovery
Teesside University is partnering with the University of Alabama at Birmingham and Pennington Biomedical Research Centre to develop personalised medicines for Neurofibromatosis Type 1 (NF1). The research focuses on creating targeted therapies tailored to the specific genetic mutations carried by individual patients.
NF1 is a complex genetic disorder with diverse mutations, making it challenging to develop effective treatments. Current therapies are limited and do not address the underlying genetic causes. The challenge was to create personalised medicines that target the specific mutations in NF1 patients.
The project employed exon-skipping techniques to develop targeted therapies for NF1. By analysing individual genetic profiles, the team aimed to create treatments that address the specific mutations present in each patient’s NF1 genes. This approach focused on restoring the function of the neurofibromin protein, which is crucial for managing the disorder.
The development of personalised medicines for NF1 could significantly improve treatment outcomes and quality of life for patients. The research aims to move towards clinical trials, offering new, tailored treatment options that address the genetic basis of NF1. This could lead to more effective therapies and potentially cures for patients with this challenging genetic disorder.
