CRISPR Partially Restores Vision to Blind Patients During Trial
Results from a Phase 1/2 clinical trial indicate that CRISPR gene-editing has enhanced the vision of individuals with inherited blindness, offering promising prospects for patients and suggesting broader applications of CRISPR in human therapeutics.
“Leber Congenital Amaurosis (LCA)” is a rare disorder affecting approximately one in every 40,000 newborns. Individuals with LCA carry a genetic mutation that severely impairs vision, leading to complete blindness in about one-third of cases. Presently, there are no FDA-approved treatments available.
The BRILLIANCE Trial and CRISPR Application
However, this scenario may soon change. “The BRILLIANCE trial has been exploring the use of CRISPR to modify the CEP290 gene, a primary contributor to LCA, in 14 patients.” This gene-editing therapy directly administers CRISPR to the light-sensitive cells behind the retina, marking the first instance of CRISPR use within the human body.
We have outlined the initial results of the trial, which span from 2020 to 2023. We assessed each participant’s treatment in one eye and then evaluated them across four parameters: “object and letter recognition on a chart, perception of colored light points in a full-field test, navigation of a maze under various light conditions, and self-reported changes in quality of life.”
Out of the 14 participants, 11 (79%) exhibited improvements in at least one of these aspects, with six (43%) showing enhancements in two or more areas. Six individuals also reported enhanced quality of life due to vision improvements, while four (29%) demonstrated clinically significant progress in eye chart tests.
Physician’s Perspective and Impact on Patients
Mark Pennesi, the study’s corresponding author, expressed the profound impact of vision improvements on patients’ daily lives, citing examples such as “locating misplaced items and discerning appliance status indicators.” While seemingly mundane tasks to those with normal vision, these improvements hold immense significance for individuals with impaired vision.
The study reported no serious adverse effects, with any mild to moderate issues promptly resolved. These findings suggest that CRISPR holds promise not only for treating LCA but potentially for addressing other forms of blindness or genetic disorders.
Read the Original Article on: New Atlas
Read more: CRISPR Test On Monkeys And No Off-target Mutations
