By In a groundbreaking move, scientists used CRISPR-Cas9 to restore vision in individuals with inherited or congenital blindness. A clinical trial showed 11 out of 14 participants gained improved vision without serious side effects.
Promising Results for Inherited Blindness
Lead researcher Eric Pierce from Harvard Medical School expressed satisfaction with the outcomes, stating the impact on participants’ lives was profound.
Study Overview: BRILLIANCE
Published in The New England Journal of Medicine on May 6, 2024, the “BRILLIANCE” study focused on individuals with Leber Congenital Amaurosis (LCA), a rare inherited blindness condition.
CRISPR Gene Therapy: EDIT-101
Participants received a single dose of CRISPR gene therapy named EDIT-101, aiming to correct the mutation in the CEP290 gene and restore normal protein function.
Visual Assessment Post-Treatment
Participants’ visual abilities were assessed post-treatment, including their perception of colored lights, navigation in varying light conditions, and reading from a chart. Most participants showed visual improvement, with six experiencing significant enhancements in quality of life related to vision.
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Favorable Safety Profile
EDIT-101 exhibited a favorable safety profile, with no serious adverse effects reported. Any mild adverse effects were transient and swiftly resolved.
CRISPR’s Potential in Genetic Conditions
The success of the BRILLIANCE study underscores CRISPR’s potential in addressing genetic conditions. Over two hundred individuals have undergone experimental CRISPR treatments, advancing towards broader clinical applications beyond approved treatments. Ongoing trials explore CRISPR therapies for diverse conditions, promising a new era in genome editing technologies.
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