Biodiscovery
Teesside University is partnering with the University of Alabama at Birmingham and 4basebio to develop a gene therapy for Neurofibromatosis Type 1 (NF1). The project aims to create a universal therapy that can address the genetic defects underlying NF1, providing a more effective treatment option for patients.
NF1 is a genetic disorder with limited treatment options with significant side effects from current therapies. The challenge was to develop a gene therapy that corrects the genetic defect responsible for NF1 directly and could be applied to all patients with the condition.
The team developed lipid-based carriers for delivering the therapeutic genes and employed a patented process to produce long DNA sequences needed for effective treatment. By focusing on a universal approach, the project aims to create a therapy that can be used broadly across different genetic mutations of NF1.
The research has the potential to revolutionise the treatment of NF1 by providing a gene therapy applicable to all patients. This could lead to significant improvements in patient outcomes, reducing the need for current treatments and offering a more targeted and effective solution. The project’s success could also pave the way for similar approaches in treating other genetic disorders.
