Organoids test gene therapy for rare eye disease
Alternatives to Animal Testing, Experimentation and Dissection - An Animal Rights Article from All-Creatures.org

FROM CAARE Citizens for Alternatives to Animal Research
February 2021

Instead of blinding animals, retina-like structures were made from cells of human patients.

baby's eye

Leber congenital amaurosis is a rare genetic disease that causes blindness in children.

Rather than using flawed and cruel methods that blind animals, scientists developed retinal organoids, or miniature retina-like structures made from cells of patients and their unaffected family members.

They found that gene augmentation (adding an extra copy of the normal gene) indicated improved vision in the afflicted organoids, opening the door to new gene therapies.

From NIH: Researchers use patients’ cells to test gene therapy for rare eye disease

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