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Melbourne scientists develop RNA therapy to save children's sight from Usher syndrome

New approach overcomes gene size barrier that has blocked conventional treatment

By LineZotpaper
Published
Read Time2 min
Sources3 outlets
Melbourne researchers are developing a treatment they hope will prevent the progressive blindness caused by Usher syndrome, a genetic condition that is the leading cause of deafblindness in Australia. The therapy, called EDITRA, aims to correct the genetic error behind one variant of the syndrome by repairing the cell's RNA instructions rather than replacing the faulty gene itself.

Poppy Stevens was just a few months old when genetic testing confirmed she had Usher syndrome, after she failed her newborn hearing test. The diagnosis meant the five-year-old, who now uses a hearing aid, could also progressively lose her vision during adolescence and early adulthood.

Her mother Ali Stevens, who lives in Melbourne's south-east, described the news as a second wave of devastation.

"Suddenly I'm thinking, 'is my child going to be blind?' We were going through the worst-case scenarios ... Is she going to work? Is she going to drive? Will this make it harder for her to have a family? Is she going to live with us for the rest of her life?" she said.

Usher syndrome affects about one in 6000 children and is the primary cause of deafblindness in Australia. Children are typically born with hearing loss and then lose their vision progressively.

At the Centre for Eye Research Australia, managing director Professor Keith Martin and Professor Guei-Sheung (Rick) Liu are creating EDITRA, a treatment that aims to correct the tiny spelling mistake in a person's genetic code that causes Poppy's variant, Usher syndrome type 2A.

The project, developed by the centre's new spin-out company Adara Bio, addresses a major barrier to treatment: the mutated genes behind Usher syndrome are too large to fit into current gene therapies. Instead of replacing the large gene, the treatment fixes the cell's genetic instructions, known as its RNA, allowing it to fit inside a single modified virus that is injected into the eye.

The work is among stories featured ahead of the Melbourne Assembly civic forum taking place next week.

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Analysis

Why This Matters

  • Usher syndrome is the leading cause of deafblindness in Australia, affecting about one in 6000 children, who typically lose vision in adolescence and early adulthood.
  • Conventional gene therapy has been blocked for these patients because the mutated genes are too large to fit inside delivery viruses.
  • The RNA-based approach, if it reaches patients, could offer the first treatment option for this form of the condition.

Background

Usher syndrome is a rare inherited condition that affects both hearing and vision, and is a significant cause of deafblindness worldwide. Children born with the condition usually have hearing loss from birth and develop retinitis pigmentosa, a progressive degeneration of the retina, during their teenage years or early adulthood. Gene therapy, which delivers a corrected copy of a gene inside a modified virus, has shown promise for some inherited conditions, but the size of the genes involved in Usher syndrome has made that approach impractical, prompting researchers to explore editing RNA instructions instead.

Key Perspectives

Researchers: Professor Keith Martin and Professor Guei-Sheung (Rick) Liu at the Centre for Eye Research Australia, along with the spin-out company Adara Bio, are pursuing the RNA-correction approach because it fits within the capacity of a single modified virus injected into the eye. Families affected: Parents such as Ali Stevens are seeking relief from the anxiety of watching a child face progressive blindness, and are hopeful that a cure for vision loss can be found. Critics/Skeptics: The treatment is at a development stage through a new spin-out company, and no clinical timeline or trial results have been announced. Whether the approach translates from the laboratory to patients remains to be demonstrated.

What to Watch

  • Progress of Adara Bio's EDITRA program through preclinical and clinical development.
  • Any announcement of human trial timelines for the RNA-based therapy.
  • The Melbourne Assembly civic forum next week, where the research is being highlighted as part of discussions shaping the city's future.

Sources

Zotpaper

Written by software from the reporting listed above, scored by an automated standards desk, and published without a person reading it first. If something here is wrong, tell the editor and it will be put right.

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