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Therapy prevents batten disease-related vision loss

Gene therapy that directly targeted a specific type of neuron in the retina was able to prevent vision loss in mice with late infantile Batten disease.

The disease, which is caused by rare genetic mutations, is frequently accompanied by vision decline that can lead to blindness, but current treatments are mostly ineffective.

However, previous preclinical trials investigating treatments for the CLN3 and CLN1 variants of the disease have demonstrated a partial correction of brain pathology in mouse models.

The most recent study explored whether an adeno-associated virus (AAV)-mediated gene therapy could restore CLN6 expression in the eye, by preventing degeneration of the cells that sense light.

It was reported that the therapy was able to treat vision degeneration, but only when delivered specifically to bipolar cells, a type of neuron found in the retina.

In the trial, mice were treated with eye injections that contained a functional CLN6 gene, which was encapsulated by an engineered AAV vector capable of penetrating the retina and reaching the bipolar cells.

Retinal function was significantly improved in treated mice and the photoreceptors’ sensitivity to light was preserved up to nine months.

Treatment also prevented photoreceptor degeneration and death, and the researchers believe the therapy may be able to treat vision loss in juvenile Batten disease (CLN3), the most common form.

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