CRISPR Gene Therapy Restores Vision for 11 of 14 Participants with Inherited Blindness in Landmark Clinical Trial

Boston, Massachusetts United States of America
CRISPR gene editing restores vision for 11 of 14 participants with inherited blindness in the BRILLIANCE clinical trial.
No serious adverse side effects were reported.
Participants received EDIT-101 gene therapy treatment which uses CRISPR-Cas9 to correct the CEP290 gene mutation responsible for Leber Congenital Amaurosis (LCA).
The study is led by Eric Pierce, MD, Ph.D., of Mass Eye and Ear.
The treatment is the first to use CRISPR for children born with a form of blindness.
CRISPR Gene Therapy Restores Vision for 11 of 14 Participants with Inherited Blindness in Landmark Clinical Trial

CRISPR gene editing brings hope to people with inherited blindness

A recent clinical trial, called BRILLIANCE, has shown promising results in restoring vision for individuals with a rare form of inherited blindness known as Leber Congenital Amaurosis (LCA). The study was led by Eric Pierce, MD, Ph.D., of Mass Eye and Ear and involved 14 participants who received the gene therapy treatment EDIT-101.

The trial demonstrated that CRISPR-Cas9 gene editing is a safe and effective method for correcting the CEP290 gene mutation, which is responsible for LCA. The results showed that 11 out of the 14 participants experienced improved vision without serious adverse side effects.

The study marks an important milestone in the field of gene therapy as it is the first to use this approach to treat children born with a form of blindness. Participants reported being thrilled to be able to see food on their plates and other objects that were previously unseen.

Blindness in LCA is caused by a gene mutation that prevents the CEP290 protein from functioning properly, which is critical for sight. The participants received a single dose of CRISPR gene therapy called EDIT-101, which cuts out the mutation in CEP290 and replaces it with a healthy strand of DNA, restoring normal function of the protein and allowing the retina to detect light.

The success of this clinical trial is a significant step forward in treating inherited blindness using gene editing techniques. The findings were published in The New England Journal of Medicine on May 6, 2024.

Two other studies reported similar results around the same time, with Children's Hospital of Philadelphia physicians restoring eyesight for two children and Olivia Cook, a college student from Missouri State University, experiencing improvements in her vision after receiving the experimental gene-editing treatment. These studies further validate the potential of CRISPR gene editing as a viable solution for treating inherited blindness.

The future of CRISPR gene editing

More than two hundred people have been treated with experimental CRISPR technologies, but only one CRISPR treatment has been approved for clinical use so far - Casgevy, a treatment for sickle-cell disease. Scientists are entering a new phase in genome editing technologies where they can help and cure patients with a variety of diseases not just treat them safely. Ongoing clinical trials are testing other CRISPR therapies for HIV/AIDS, diabetes, cancers, cardiovascular diseases, and antibiotic resistance.


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Confidence

90%

Doubts
  • Are there any long-term side effects of CRISPR gene therapy that haven't been identified yet?
  • Have there been any instances where CRISPR gene therapy has not worked as intended?
  • How many participants experienced minor side effects, and what were they?

Sources

99%

The overall score is a weighted number that takes into account conflict of interest, bias, deception and other practices that undermine the credibility of the source. It is calculated as:
(Site Conflicts Of Interest + Author Conflicts Of Interest) / 2.0 * 0.2 + ArticleBiasScore * 0.20 + UniquePointsScore * 0.05 + DeceptionScore * 0.20 + ReadabilityScore * 0.05 + FallacyScore * 0.20

A score that takes into consideration the content for flow, interruptions with ads, and overt search engine optimization techniques that makes the content hard to understand

Experimental gene therapy restores some vision in patients with inherited blindness

CNN News Site: In-Depth Reporting and Analysis with Some Financial Conflicts and Sensational Language Jacqueline Howard Monday, 06 May 2024 13:06
  • Unique Points
    • College student Olivia Cook participated in a clinical trial for an experimental gene-editing treatment for her inherited retinal disorder Leber congenital amaurosis (LCA) that was not mentioned in other articles.
    • Olivia Cook received the experimental gene-editing treatment through a surgery on her left eye, which is not explicitly stated in other articles.
  • Accuracy
    No Contradictions at Time Of Publication
  • Deception (100%)
    None Found At Time Of Publication
  • Fallacies (95%)
    No formal fallacies found. Some inflammatory rhetoric and anecdotal evidence used.
    • . . . a rare type of eye disorder historically called Leber congenital amaurosis or LCA.
    • A treatment that used CRISPR was found to be safe and efficacious in improving vision among a small sample of patients with inherited blindness in the Phase 1/2 clinical trial that Cook participated in.
  • Bias (100%)
    None Found At Time Of Publication
  • Site Conflicts Of Interest (100%)
    None Found At Time Of Publication
  • Author Conflicts Of Interest (100%)
    None Found At Time Of Publication

97%

The overall score is a weighted number that takes into account conflict of interest, bias, deception and other practices that undermine the credibility of the source. It is calculated as:
(Site Conflicts Of Interest + Author Conflicts Of Interest) / 2.0 * 0.2 + ArticleBiasScore * 0.20 + UniquePointsScore * 0.05 + DeceptionScore * 0.20 + ReadabilityScore * 0.05 + FallacyScore * 0.20

A score that takes into consideration the content for flow, interruptions with ads, and overt search engine optimization techniques that makes the content hard to understand

CHOP restored eyesight for two children with one injection. It’s one more step for gene editing using CRISPR.

The Philadelphia Inquirer Abraham Gutman Monday, 06 May 2024 18:13
  • Unique Points
    • Children's Hospital of Philadelphia physicians restored eyesight in two children by performing a gene therapy technique entirely inside their eyes to correct a genetic mutation causing blindness.
    • Gene editing, used to correct a deficiency in a person’s DNA causing disease, was performed inside the eyes of children for the first time.
  • Accuracy
    • The results of the clinical trial were published in the New England Journal of Medicine.
  • Deception (95%)
    The article makes several statements that are not deceptive but do contain some elements of sensationalism and selective reporting. The author states that the children's eyesight was improved 'about 10- to 100-fold' after a single injection, but does not provide any specific data or evidence to support this claim. The author also quotes Tomas Aleman stating that 'it's the first time that gene editing has been used in the eyes of children, and proved safe and successful.' This statement is not deceptive on its own, but it does create a sense of excitement and importance around the research without providing any context or perspective. The author also selectively reports on the results of the clinical trial, focusing only on the safety and vision improvement in half of the patients, while ignoring the fact that all patients had Leber Congenital Amaurosis and that some did not experience vision improvement. Additionally, there are several instances of emotional manipulation through phrases such as 'restored eyesight' and 'first-of-its-kind treatment for kids.'
    • It’s the first time that gene editing has been used in the eyes of children, and proved safe and successful.
    • Children’s Hospital of Philadelphia physicians restored eyesight in two children by performing a gene therapy technique entirely inside their eyes to correct a genetic mutation causing blindness, a first-of-its-kind treatment for kids.
    • The 10-year-old and 14-year-old patients had their eyesight improved about 10- to 100-fold after a single injection to their retina.
    • The two children were part of a clinical trial, involving hospitals in five cities, that also included 12 adults. All of the patients had Leber Congenital Amaurosis, a common cause of blindness in children.
  • Fallacies (95%)
    The article contains an appeal to authority from Eric Kmiec when he states 'Gene therapies a decade ago could only fix the error by injecting corrected DNA next to it within the cell, like striking out a word and writing it the right way by its side.' and 'That approach has a few problems, Kmiec said. The body can reject the corrected cells like any transplantation. And cells in the nervous system don’t multiply.' However, there are no explicit dichotomous depictions or inflammatory rhetoric in the article.
    • ]Gene therapies a decade ago could only fix the error by injecting corrected DNA next to it within the cell, like striking out a word and writing it the right way by its side.[
    • The body can reject the corrected cells like any transplantation.
  • Bias (100%)
    None Found At Time Of Publication
  • Site Conflicts Of Interest (100%)
    None Found At Time Of Publication
  • Author Conflicts Of Interest (100%)
    None Found At Time Of Publication

99%

The overall score is a weighted number that takes into account conflict of interest, bias, deception and other practices that undermine the credibility of the source. It is calculated as:
(Site Conflicts Of Interest + Author Conflicts Of Interest) / 2.0 * 0.2 + ArticleBiasScore * 0.20 + UniquePointsScore * 0.05 + DeceptionScore * 0.20 + ReadabilityScore * 0.05 + FallacyScore * 0.20

A score that takes into consideration the content for flow, interruptions with ads, and overt search engine optimization techniques that makes the content hard to understand

CRISPR gene editing trial treats inherited blindness

Deutsche Welle News Site Review by AI - Bias, Contradictions, Deceptiveness Analysis (English Edition) Fred Schwaller Monday, 06 May 2024 18:15
  • Unique Points
    • Scientists used CRISPR-Cas9 to restore vision in people with a rare form of inherited or congenital blindness.
    • 11 out of 14 participants in the clinical trial experienced improved vision without serious adverse side effects.
    • The study was the first to use gene therapy to treat children born with a form of blindness.
    • Harvard Medical School’s Eric Pierce led the study, and participants reported being thrilled to be able to see food on their plates.
    • Blindness in LCA is caused by a gene mutation that prevents the CEP290 protein from functioning properly, which is critical for sight.
    • Participants received a single dose of CRISPR gene therapy called EDIT-101, which cuts out the mutation in CEP290 and replaces it with a healthy strand of DNA, restoring normal function of the protein and allowing the retina to detect light.
    • Emmanuelle Charpentier and Jennifer A. Doudna were awarded the 2020 Chemistry Nobel Prize for discovering CRISPR-cas9.
    • In the BRILLIANCE study, participants showed visual improvements in color recognition, maze navigation, and reading from a chart after receiving the treatment.
    • Almost all participants (except three) showed some level of visual improvement; six had major improvements in vision-related quality of life and could identify objects and letters on a chart.
    • EDIT-101 caused no serious adverse side effects; some patients reported mild adverse effects that resolved quickly.
    • Only one CRISPR treatment, Casgevy for sickle-cell disease, has been approved for clinical use so far.
  • Accuracy
    • 11 out of 14 participants experienced improved vision without serious adverse side effects.
    • The study was the first to use gene therapy to treat children born with a form of blindness.
    • Participants received a single dose of CRISPR gene therapy called EDIT-101, which cuts out the mutation in CEP290 and replaces it with a healthy strand of DNA, restoring normal function of the protein and allowing the retina to detect light.
    • Inherited retinal degenerations are a leading cause of blindness around the world.
    • The gene-editing tool CRISPR was found to be safe and efficacious in improving vision among most patients in the clinical trial.
  • Deception (100%)
    None Found At Time Of Publication
  • Fallacies (100%)
    None Found At Time Of Publication
  • Bias (100%)
    None Found At Time Of Publication
  • Site Conflicts Of Interest (100%)
    None Found At Time Of Publication
  • Author Conflicts Of Interest (100%)
    None Found At Time Of Publication

100%

The overall score is a weighted number that takes into account conflict of interest, bias, deception and other practices that undermine the credibility of the source. It is calculated as:
(Site Conflicts Of Interest + Author Conflicts Of Interest) / 2.0 * 0.2 + ArticleBiasScore * 0.20 + UniquePointsScore * 0.05 + DeceptionScore * 0.20 + ReadabilityScore * 0.05 + FallacyScore * 0.20

A score that takes into consideration the content for flow, interruptions with ads, and overt search engine optimization techniques that makes the content hard to understand

CRIPSR gene editing leads to improvements in vision for people with inherited blindness, clinical trial shows

MedicalXpress Science X Monday, 06 May 2024 18:18
  • Unique Points
    • The BRILLIANCE clinical trial shows CRISPR gene editing is safe and led to measurable improvements in 11 of the 14 participants with a form of inherited blindness caused by mutations in the CEP290 gene.
    • Six participants experienced meaningful improvements in cone-mediated vision, which is used for daytime and central vision.
    • The genome editing company Editas Medicine began exploring how to tackle the CEP290 mutation in 2014, conducting preclinical studies to determine whether a gene editing approach like CRISPR-Cas9 might be feasible to target these large gene mutations.
    • Exploring CRISPR as an inherited retinal disorder treatment: Mutations in the CEP290 gene are the leading cause of inherited blindness taking place during the first decade of life. The mutations cause rod and cone photoceptors in the eye’s retina to function improperly, which after some time will lead to irreversible vision loss.
    • The first patient to receive a CRISPR treatment inside the body (in vivo) took place at the Casey Eye Institute at Oregon Health & Science University (OHSU), under the leadership of Mark Pennesi, MD, Ph.D.
    • Additional participants were treated across three other trial sites: Bascom Palmer Eye Institute, W.K. Kellogg Eye Center, and Scheie Eye Institute at the Children’s Hospital of Philadelphia (CHOP) and the Hospital of the University of Pennsylvania.
  • Accuracy
    No Contradictions at Time Of Publication
  • Deception (100%)
    None Found At Time Of Publication
  • Fallacies (100%)
    None Found At Time Of Publication
  • Bias (100%)
    None Found At Time Of Publication
  • Site Conflicts Of Interest (100%)
    None Found At Time Of Publication
  • Author Conflicts Of Interest (100%)
    None Found At Time Of Publication

100%

The overall score is a weighted number that takes into account conflict of interest, bias, deception and other practices that undermine the credibility of the source. It is calculated as:
(Site Conflicts Of Interest + Author Conflicts Of Interest) / 2.0 * 0.2 + ArticleBiasScore * 0.20 + UniquePointsScore * 0.05 + DeceptionScore * 0.20 + ReadabilityScore * 0.05 + FallacyScore * 0.20

A score that takes into consideration the content for flow, interruptions with ads, and overt search engine optimization techniques that makes the content hard to understand

Experimental gene therapy restores some vision in patients with inherited blindness

WRAL News Unknown WRAL Monday, 06 May 2024 13:06
  • Unique Points
    • Olivia Cook was able to see her friends' faces glow under twinkling Christmas lights in dim lighting after receiving the experimental gene-editing treatment.
    • The therapy remains experimental and the results need to be replicated in a larger group of people.
    • Children's Hospital of Philadelphia physicians restored eyesight in two children by performing a gene therapy technique entirely inside their eyes to correct a genetic mutation causing blindness.
  • Accuracy
    No Contradictions at Time Of Publication
  • Deception (100%)
    None Found At Time Of Publication
  • Fallacies (100%)
    None Found At Time Of Publication
  • Bias (100%)
    None Found At Time Of Publication
  • Site Conflicts Of Interest (100%)
    None Found At Time Of Publication
  • Author Conflicts Of Interest (100%)
    None Found At Time Of Publication