Waterford research will enhance therapy of rare disease
Waterford Institute of Technology (WIT) is to begin developing enhanced gene therapy treatments for the rare human disorder Canavan disease. WIT will collaborate with the University of Massachusetts Medical School (UMASS) in leading this exciting new PhD project.
Canavan disease (CD) is caused by a mutation in one gene, which leads to the loss of an enzyme (ASPA, a protein which carries out reactions in the body) and consequently a build-up of a chemical in the brain. Symptoms of CD include intellectual disability, loss of motor skills, abnormal muscle tone, and visual degeneration, with most CD patients dying in childhood.
Leading this project is Dr Lee Coffey, a lecturer and researcher in the Science Department in WIT. Dr Coffey leads the Molecular Biotechnology and Biopharmaceutical Research Group which is part of the PMBRC. The research group has demonstrated success in researching various genes and enzymes, answering needs in industrial and biopharmaceutical spaces, with numerous publications and patent applications under its belt.
Dr Coffey expanded the focus of the group to target genes/enzymes associated with rare diseases that are not addressed by pharmaceutical companies. Other WIT researchers to be involved in this project include Dr Laurence Fitzhenry and Dr Tríona Dooley-Cullinane, who also have extensive research and commercialisation experience in the biotechnology/biomedical activities at WIT.
A PhD candidate on WIT’s structured PhD programme will also work on the research. The PhD position is funded by the WIT PhD Scholarship Programme for a 4-year research project and applications are open until 26 April.
Dr Coffey said: “I’m delighted to see this project about to become realised and to see WIT involved with world-class researchers at UMASS. This project will involve advanced skills and topics that are becoming very relevant in the biopharmaceutical sector, which are also covered well in BSc Hons in Molecular Biology with Biopharmaceutical Science delivered at WIT.”
Professor Guangping Gao of UMASS will co-supervise this project and has extensively researched CD and potential gene therapy solutions for many years. This work has resulted in the successful treatment of mice with CD as disease models. Prof Gao is the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research. Prof Gao was also recently ranked one of the world’s top translational researchers by Nature Biotechnology.
This project aims to develop an enhanced ASPA protein using molecular biology techniques at a genetic level, with in-vitro testing at WIT and in-vivo gene therapy testing against disease models in UMASS. Gene therapy treatments already under development in Prof Gao’s lab may benefit from an enhanced gene being delivered to CD sufferers, meaning that the resulting enhanced ASPA enzyme may treat CD more efficiently. It is hoped that this research will inform an enhanced gene therapy for future human CD patient treatments.
Applications are invited for this PhD position funded by the WIT PhD Scholarship Programme, for a 4-year research project. The candidate is expected to be self-motivated, capable of working on his or her own initiative and fit in with the friendly atmosphere within the wider research team.
The candidate will be enrolled on the WIT structured PhD programme and will have the opportunity to complete a short secondment to the US-based UMASS/Horae Gene Therapy Center with co-supervisor Prof Guangping Gao to analyse potentially enhanced gene therapies using disease models.
The PhD project will cover fees in addition to a personal stipend and research expenses. Find more information and how to apply before the 26 April 2019 closing date here.