Chinese Researchers Notch Major Milestone in Developing Gene Editing System Against Autism

Chinese researchers may have scored a major win in the fight against autism after they successfully edited a mutated gene associated with autism in mice. The research team developed a genome-editing system capable of treating ASD-related mutations in mice using base editing in the brain.

This treatment represents the first-ever injection with the ability to reverse autism symptoms, at least in mouse models, and could open the door to the development of alternative treatments for other neurodevelopmental disorders. The injection corrects mutations in the MEF2C gene, which typically occur in patients with autism spectrum disorder (ASD).

Estimates from the United States Centers for Disease Control and Prevention (CDC) show that around 1% of the global population and 1 in every 36 children in the United States has autism spectrum disorder. The neurodevelopmental condition is characterized by symptoms such as repetitive behaviors, difficulty communicating and interacting with others, and intense interest.

Depending on the severity of symptoms and the presence or absence of adequate coping mechanisms, autism spectrum disorder patients may find it difficult or nearly impossible take part in day-to-day activities that involve social interaction.  Interestingly, many of the symptoms caused by MEF2C mutations have been linked to other neurological disorders such as epilepsy.

Current treatments for autism can help patients regulate their behavior, but there is no cure for the condition.

According to the Chinese research team’s paper, the gene editing system treated the mutations by restoring MEF2C protein levels in different regions of the brain and ultimately “reversed the behavioral abnormalities” in mice with the MEF2C mutation. Researchers believe this mutation is “strongly associated” with autism spectrum disorder and contributes to symptoms such as speech difficulties, development deficits and repetitive behaviors.

The treatment was provided via a simple injection into a vein in the tail, and researchers from several Chinese institutions observed the mice for weeks to track changes in their behavior.

The researchers believe the new injection-based, gene-editing system may have applications in other conditions as well. They say individualized gene-editing therapy to treat autism spectrum disorder and other neurodevelopmental and neurological disorders could be a feasible and affordable option “in the near future.”

While this treatment could be effective at treating patients with conditions caused by the MEF2C mutation, it may not be suitable for people with conditions caused by complex mutations rather than single nucleotide variations.

The researchers published their findings in the “Nature Neuroscience” journal on Nov. 27, 2023.

This positive step in gene editing to address the autism challenge comes at a time when many drug manufacturers such as PaxMedica Inc. (NASDAQ: PXMD) are working to develop effective treatments to combat this condition that hampers the quality of life of patients around the world.

NOTE TO INVESTORS: The latest news and updates relating to PaxMedica Inc. (NASDAQ: PXMD) are available in the company’s newsroom at https://ibn.fm/PXMD

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