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Nobel Prize-winning CRISPR/Cas9 technology of major significance for blindness treatment

10 October 2020

CRISPR/Cas9 genetic scissors have been of great importance to researchers at St. Erik Eye Hospital and Karolinska Institutet for refining the production of retinal cells from embryonic stem cells for the treatment of blindness in elderly. Yesterday, the Royal Swedish Academy of Sciences decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier and Jennifer A. Doudna for the discovery of CRISPR technology. 

anders-kvanta

Professor and Senior Consultant Anders Kvanta.

Researchers at St. Erik Eye Hospital and Karolinska Institutet have used CRISPR/Cas9 genetic scissors to successfully produce retinal cells aimed for treatment of age-related macular degeneration (AMD), the most common cause of blindness in elderly. CRISPR technology has made it possible to modify so-called hESC-RPE cells so that they evade the immune system, which in turn prevents rejection following transplantation.

Anders Kvanta, Senior Consultant at St. Erik Eye Hospital and Professor at Karolinska Institutet, who together with researcher Fredrik Lanner at Karolinska Institutet is responsible for the research, is extremely pleased that the CRISPR technology has now been acknowledged in this way.

First and foremost, this technology heralds an amazing breakthrough in our opportunities to genetically tailor-make cells for different purposes. Our research is focused on developing therapies for hereditary and age-related blindness where retinal cells die. CRISPR technology enables the production of retinal cells that are not rejected by the immune system following transplantation, something we have already demonstrated in animal models. This in turn means that the patients who are treated with these cells will not need to take immunosuppressive drugs, he says.

According to Anders Kvanta, in the future CRISPR technology will most probably enable genetic correction  of retinal cells, thereby preventing hereditary blindness.


Text: Helena Mayer

Updated
16 October 2020