Stem Cell-Derived Exosomes for the Treatment of Glaucoma

Brief plain language background

Glaucoma is the leading cause of irreversible blindness and is characterised by damage to cells at the back of the eye, known as retinal ganglion cells (RGC), which extend into the optic nerve. RGCs convert light into signals that pass along the optic nerve – connecting the eye to the brain.

Around 80 million people across the globe have glaucoma, and with the ageing population, this number is projected to increase to 120 million by 2040.

What problem/knowledge gap does it help address

A loss of function of RGCs means incoming light signals cannot be shared with the brain, known as a neurodegeneration.

A risk factor for RGC loss is increased intraocular pressure and treatment for glaucoma tends to focus on reducing this. However, the condition often progresses despite the treatment to reduce intraocular pressure, suggesting a need for an additional treatment that protects RGCs where currently, none exists.

Aim of the project

Exosomes are small vesicles (fluid filled sacs) containing small amounts of molecules found in cells and genetic material known as miRNA. Exosomes are released and can be absorbed between nearby cells. Stem cells – cells with potential to develop into different specialised types of cells – also release exosomes, which could be utilised to deliver materials directly to non-functioning cells.

This project aims to test exosomes, isolated from a range of stem cells, as therapy for glaucoma.

Key procedures/objectives

1. Isolate exosomes from stem cells sourced from a range of human tissues.
2. Investigate which exosomes have therapeutic potential when tested on a rodent model of the retina, utilising these findings to test the best candidates with a rodent model of glaucoma.
3. Test the candidate exosomes in a model of human RGC death.

Potential impact on people with sight loss

Currently untreatable, glaucoma leads to irreversible damage to RGCs and the optic nerve. Treatment that focuses on preventing or reversing damage to nerve cells (neuroprotection) has the potential to slow down or stop sight loss for people living with glaucoma. If successful, finding candidate exosomes from a range of stem cells has the potential to be developed into neuroprotective treatments for glaucoma, where currently none exist.