Researchers Correct CRISPR Study on Treating Rare Neurodisorders

Researchers at the University of California San Francisco have issued a correction to a recent study published in the journal Nature, which explored the use of CRISPR activation for treating SCN2A-related neurodevelopmental disorders. The correction, made on September 17, 2025, involves a minor adjustment to a figure in the original article. According to the correction notice, the exons in the top line of Fig. 1b, which depicts the Scn2a allele, were initially flipped and read as 3 4 5. The corrected version now reads 5 4 3, as reflected in both the HTML and PDF versions of the article. The correction was made to ensure the accuracy of the scientific representation. The original study, published on September 17, 2025, aimed to investigate the potential of CRISPR activation as a therapeutic approach for SCN2A-related neurodevelopmental disorders, including autism spectrum disorders and channelopathies. The research team, led by Serena Tamura, Andrew D. Nelson, and Perry W. E. Spratt, employed CRISPR activation to modulate gene expression in patient-derived cells and demonstrated promising results. Dr. Tamura, a researcher at the University of California San Francisco, acknowledged the importance of accuracy in scientific representation. "We strive to maintain the highest standards of scientific integrity, and this correction is a testament to our commitment to transparency and accountability," she said. SCN2A-related neurodevelopmental disorders are a group of genetic conditions characterized by developmental delays, intellectual disability, and seizures. The disorders are caused by mutations in the SCN2A gene, which encodes a sodium channel protein essential for normal brain function. The correction does not affect the overall findings or conclusions of the original study, which highlighted the potential of CRISPR activation as a therapeutic strategy for SCN2A-related disorders. The research team plans to continue exploring the therapeutic potential of CRISPR activation and to investigate its safety and efficacy in preclinical models. The correction serves as a reminder of the importance of accuracy and attention to detail in scientific research. As researchers continue to push the boundaries of genetic engineering and gene therapy, the need for rigorous quality control and transparency is essential to advancing our understanding of these complex disorders and developing effective treatments.