Using tiny worms to identify gene therapies for age-related diseases

Using the nematode worm C.elegans as a model to identify gene therapies for age-related neurodegenerative diseases

In this project uniQure and researchers of ERIBA, UMCG, explore the rapidly ageing, small nematode worm C.elegans as a model to accelerate the identification of candidate genes and siRNA’s for gene therapy to treat age-related neurodegenerative diseases.

Due to the increasing life-expectancy of our population, age-related neurodegenerative diseases like Parkinson’s and Alzheimer’s disease, will have a growing impact on our society, economy, and healthcare systems. The number of people suffering from such diseases is expected to increase to over a 100 million world-wide by 2050. There is no cure for these diseases and their cause is still incompletely understood.

The development of an effective therapy for such age-related diseases requires long, low-throughput, and expensive studies in human cell-based models and mouse models. In order to accelerate the identification of targets and the exploration of therapeutic interventions, it will be important to develop fast screening tools and models. The small nematode worm C.elegans has been extensively used as a screening model for biomedical research and drug discovery. This worm has a very short lifespan of around three weeks and, because of its small size (1 mm), allows for high throughput studies at low costs.

Researchers from the research group of Ellen Nollen at ERIBA, UMCG, have a longstanding expertise in C.elegans research and genetic screening. uniQure is specialized in the development of gene therapies, including for neurodegenerative diseases.

Through their collaboration, uniQure and ERIBA hope to develop C.elegans models and a high-throughput pipeline that can be used to identify and select candidate target genes and test therapeutic RNA’s. If successful, they hope that this project may accelerate and reduce costs to study disease mechanisms and develop therapies for age-related neurodegenerative diseases.

Summary

The development of an effective gene therapy requires long, low-throughput, and expensive studies in human cells and mouse models. In particular for age-related diseases such studies may be challenging and costly, as disease symptoms will only occur at old age. In this project they will explore the rapidly ageing, small nematode worm C.elegans as a model for high-throughput screening for candidate genes and RNA’s for gene therapy.

Time period

12 months

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