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Tag: CRISPR

  • CRISPR Partially Restores Vision to Blind Patients During Trial

    CRISPR Partially Restores Vision to Blind Patients During Trial

    CRISPR restores vision in patients with hereditary blindness in a new trial. Credit: Freepik
    CRISPR restores vision in patients with hereditary blindness in a new trial. Credit: Freepik

    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

  • CRISPR Test On Monkeys And No Off-target Mutations

    CRISPR Test On Monkeys And No Off-target Mutations

    In the initial comprehensive study conducted on monkeys, it was discovered that CRISPR does not induce unintended off-target mutations.
    In the initial comprehensive study conducted on monkeys, it was discovered that CRISPR does not induce unintended off-target mutations. Credit : galitskaya/Depositphotos.

    As the first human trials utilizing the potentially groundbreaking CRISPR gene editing technique are set to begin in the UK and the US, there are lingering concerns regarding the overall safety of this system. To address these concerns, a group of Chinese researchers has conducted a comprehensive evaluation of the impact of CRISPR-Cas9 on non-human primates, aiming to determine if it leads to off-target mutations.

    Last year, a contentious study proposed that CRISPR could introduce unintended mutations. However, due to numerous criticisms from scientists regarding the reliability of the data, the study was eventually retracted.

    Although the previously flawed research has been retracted, significant apprehensions persist regarding the possibility of unintended mutations arising from gene editing using the CRISPR technique. The recent Chinese study represents the first comprehensive investigation that examines the relationship between the CRISPR process and de novo mutations, which are newly occurring mutations that arise spontaneously in a fertilized egg during the initial stages of embryogenesis, specifically in rhesus macaque monkeys.

    Employing The System

    The study was initiated by employing the CRISPR system on macaque zygotes, which are fertilized eggs. The primary objective of the researchers was to assess the targeting specificity of the technique by specifically targeting the deletion of a gene called MCPH1, known to be associated with birth defects in humans. In the embryos that underwent the initial treatment, the CRISPR process effectively eliminated the MCPH1 gene in 13 out of the 15 samples, showcasing successful results.

    As a result, a total of five monkeys were eventually gestated, and four of them successfully survived delivery. All of the offspring monkeys exhibited the desired CRISPR modifications. However, the subsequent course of action involved conducting thorough whole genome sequencing to gain a deeper understanding of whether the gene-editing process had led to unintended mutations.

    Read The Original Article: Chinese scientists test CRISPR on Monkeys and find no off-target mutations

    Read More: A Spider Monkey’s Remains Tell a Story of Old Diplomacy in the Americas