CRISPR gibt blinden Patienten in einer klinischen Studie einen Teil des Sehvermögens zurück | Die Ergebnisse geben Patienten mit dieser Erkrankung nicht nur neue Hoffnung, sondern zeigen auch, dass CRISPR beim Menschen zur Behandlung einer Reihe von Erkrankungen eingesetzt werden könnte.

From the article: CRISPR gene-editing has improved the vision of patients with an inherited form of blindness, according to results of a Phase 1/2 clinical trial. The results not only give new hope to patients with the condition, but show that CRISPR could be put to use in humans to treat a range of conditions.

Leber Congenital Amaurosis (LCA) is a rare condition that affects about one in every 40,000 newborns. Those affected have a genetic mutation that results in severely reduced vision, leading to complete blindness in about a third of patients. Currently, there are no FDA-approved treatment options at all.

But that might be about to change. The BRILLIANCE trial has been investigating using CRISPR to edit the CEP290 gene, one of the main culprits behind LCA, in 14 patients. The gene-editing treatment is delivered directly to the light-sensitive cells behind the retina, making this the first time CRISPR had been put to work directly in the human body.

Now, the team has detailed the [results](https://www.nejm.org/doi/full/10.1056/NEJMoa2309915) from the first three years of the trial, from 2020 to 2023. Each patient received the treatment in one eye, and were then monitored for four different outcomes: identifying objects and letters on a chart; how well they could see colored points of light in a full-field test; how well they could navigate a maze with physical objects and different light levels; and their own reported experiences of quality of life changes.