CRISPR Gene Editing Trial for Inherited Blindness Reveals Promising Results

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Mass Eye and Ear

Mass Eye and Ear

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Mass Eye and Ear led the BRILLIANCE trial testing an experimental CRISPR-based gene editing medicine for a form of Leber Congenital Amaurosis, a condition that is a leading cause of childhood blindness. The researchers found that the treatment was safe and led to measurable improvements in 11 of the participants treated.
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