Researchers Correct Study on CRISPR Therapy for Neurodevelopmental Disorders

Researchers behind a recent study on CRISPR activation for SCN2A-related neurodevelopmental disorders have issued a correction to their original article, published in the journal Nature on September 17, 2025. The correction pertains to a figure in the study, specifically in Fig. 1b, where the exons in the top line depicting the Scn2a allele were incorrectly labeled as 3 4 5, and have now been corrected to 5 4 3 in both the HTML and PDF versions of the article. According to the correction notice, the mistake was identified and corrected by the authors, who contributed equally to the study. Serena Tamura, Andrew D. Nelson, and Perry W. E. Spratt, among others, were involved in the research, which aimed to explore the potential of CRISPR activation as a therapeutic approach for SCN2A-related neurodevelopmental disorders, including autism spectrum disorders and channelopathies. The study's findings were significant, as they suggested that CRISPR activation could be a viable treatment option for these disorders, which are often caused by mutations in the SCN2A gene. The researchers used CRISPR-Cas9 to activate the SCN2A gene in patient-derived cells, leading to improved neuronal function and reduced symptoms of the disorders. The correction notice was issued to ensure the accuracy and integrity of the scientific record, and to prevent any potential misinterpretation of the study's findings. The authors acknowledged the importance of correcting the mistake and thanked the journal for its prompt attention to the issue. In a statement, Serena Tamura, one of the lead authors, noted that "the correction is a minor one, but it's essential to ensure the accuracy of our findings. We're committed to transparency and accountability in our research, and we appreciate the journal's diligence in addressing this issue." The study's findings have significant implications for the treatment of SCN2A-related neurodevelopmental disorders, and the correction notice does not affect the overall validity or significance of the research. The journal's decision to publish the correction notice demonstrates its commitment to maintaining the highest standards of scientific integrity and accuracy. As research continues to advance in the field of gene regulation and CRISPR technology, scientists and clinicians are working to translate these findings into effective treatments for patients with SCN2A-related disorders. The correction notice serves as a reminder of the importance of attention to detail and the need for ongoing scrutiny and evaluation of scientific research. The study's authors are currently working on further research to explore the potential of CRISPR activation as a therapeutic approach for SCN2A-related disorders. The journal has not announced any plans to retract the original article, and the study's findings remain a significant contribution to the field of neurodevelopmental disorders research.