CRISPR Breakthrough Corrected: UCSF Researchers Refine Treatment for Neurodevelopmental Disorders

Researchers at the University of California San Francisco have issued a correction to a recent study published in the journal Nature, detailing a breakthrough in using CRISPR activation to treat SCN2A-related neurodevelopmental disorders. The correction, made on September 17, 2025, involves a minor error in a figure depicting the Scn2a allele, which has been corrected to reflect the accurate sequence of exons. According to the correction notice, in the initial version of the article, Fig. 1b showed the exons in the top line of the Scn2a allele as 3 4 5, which has now been corrected to 5 4 3 in both the HTML and PDF versions of the article. The correction was made to ensure the accuracy of the scientific data presented in the study. The study, which was published on September 17, 2025, aimed to explore the use of CRISPR activation as a potential treatment for SCN2A-related neurodevelopmental disorders, including autism spectrum disorders and channelopathies. The researchers, led by Serena Tamura, Andrew D. Nelson, and Perry W. E. Spratt, used a combination of CRISPR activation and gene regulation techniques to develop a novel therapeutic approach for these disorders. In an interview, Serena Tamura, one of the lead authors of the study, emphasized the importance of accuracy in scientific research. "We take great care to ensure the accuracy of our data, and we appreciate the opportunity to correct this minor error," she said. "Our study highlights the potential of CRISPR activation as a treatment for SCN2A-related neurodevelopmental disorders, and we look forward to continuing our research in this area." SCN2A-related neurodevelopmental disorders are a group of genetic disorders caused by mutations in the SCN2A gene, which codes for a sodium channel protein. These disorders can result in a range of symptoms, including autism spectrum disorders, epilepsy, and developmental delays. The study's findings suggest that CRISPR activation may be a promising therapeutic approach for treating these disorders. The correction to the study does not affect the overall conclusions or findings of the research, and the study remains a significant contribution to the field of neurodevelopmental disorders. As research continues to advance, scientists and clinicians are hopeful that CRISPR activation may one day become a viable treatment option for patients with SCN2A-related neurodevelopmental disorders. The study's authors are currently working to further develop and refine their CRISPR activation technique, with the goal of translating their findings into clinical trials. As the field of gene regulation and CRISPR technology continues to evolve, researchers are optimistic about the potential for breakthroughs in treating a range of genetic disorders.