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Hope for previously incurable diseases

Gene therapy for inherited retinal diseases is being validated in several clinical trials and is becoming a promising therapeutic option

Dermot McGrath

Posted: Tuesday, September 12, 2017

Dominik Fischer

Gene therapy for inherited retinal diseases is currently being validated in several clinical trials and is becoming a promising therapeutic option for these previously incurable diseases, Dominik Fischer MD said at the Young Retinal Specialist (YOURS) Day.

In a review of the history and rationale for gene therapy, Dr Fischer explained that the retina is made up of different cell populations such as retinal pigment epithelium (RPE), photoreceptors and ganglion cells.

“The retina is uniquely susceptible to genetic mutations. A lot can go wrong genetically speaking and there are a whole host of mechanisms that can be targeted by genetic mutations and can lead to blindness. So these genetic mutations can potentially be addressed by gene therapy and this is why it deserves interest,” he said.

Gene therapy can work well in retinal disease stages where there is still photoreceptor function, said Dr Fischer, but it is less likely to deliver any benefit in late-stage retinal diseases where neither photoreceptor nor RPE cells are available to be transduced by gene therapy. In these cases, strategies such as electronic implants or stem cell therapy are more likely to prove beneficial, he said.

The history of gene therapy stems back to the 1940s and 1950s when researchers came to realise that it is actually the DNA that transports information that is useful and necessary for cells to have a physiological potential, said Dr Fischer. Later on in the 1960s investigators discovered that viral infections, such as that of the uterus for example, can lead to cancer.

“They were puzzled as to how a virus could cause cancer until it was found that it was actually due to the DNA contained in the virus that was transforming the cell function,” he said.