NEW DELHI: In what could be a major breakthrough in the field of medical science, Chinese scientists have created the first injection that can reverse autism symptoms through genetic base editing within the brain, reported the South China Morning Post.
The Shanghai-based researchers have found positive results from the test conducted on mice.
The researchers reportedly developed a genome editing system that effectively altered the DNA of mice carrying a mutation observed in certain individuals with autismspectrum disorder (ASD).Mice receiving the injection containing this editing system exhibited a reduction in behaviours associated with ASD.
In a research paper published on November 27 in the journal Nature Neuroscience, the scientists highlighted that the potential treatment method they discovered may not only be applicable to patients with autism spectrum disorder (ASD) but also to individuals with other genetic neurodevelopmental disorders.
ASD impacts approximately 1 per cent of the global population. According to the US Centers for Disease Control and Prevention (CDC), one in every 36 children in the United States is diagnosed with this disorder.
The disorder can hinder a person’s capacity to interact and communicate, leading to repetitive behaviours and intense interests.
In investigating the potential of genomic editing for treating ASD, the scientists generated mice with mutations in the MEF2C gene, a gene they identified as “strongly associated” with the disorder.
According to the paper, reported by the SCMP, mutations in this gene were believed to contribute to developmental deficits, speech difficulties, repetitive behaviours, and epilepsy.
Li Dali, a professor of Life Sciences at East China Normal University, who is not an author of the paper, said “This was the first effective treatment of mice with ASD-related mutations using base editing in the brain, according to Shenzhen-based autism media platform Dami & Xiaomi”.
“The treatment successfully restored MEF2C protein levels in several brain regions and reversed the behavioural abnormalities in MEF2C-mutant mice,” the paper said.
However, for some patients, the disorder may not be caused by a single nucleotide variation like it was for the mice in the study, but rather by more complex mutations that would be difficult to edit.
The Shanghai-based researchers have found positive results from the test conducted on mice.
The researchers reportedly developed a genome editing system that effectively altered the DNA of mice carrying a mutation observed in certain individuals with autismspectrum disorder (ASD).Mice receiving the injection containing this editing system exhibited a reduction in behaviours associated with ASD.
In a research paper published on November 27 in the journal Nature Neuroscience, the scientists highlighted that the potential treatment method they discovered may not only be applicable to patients with autism spectrum disorder (ASD) but also to individuals with other genetic neurodevelopmental disorders.
ASD impacts approximately 1 per cent of the global population. According to the US Centers for Disease Control and Prevention (CDC), one in every 36 children in the United States is diagnosed with this disorder.
The disorder can hinder a person’s capacity to interact and communicate, leading to repetitive behaviours and intense interests.
In investigating the potential of genomic editing for treating ASD, the scientists generated mice with mutations in the MEF2C gene, a gene they identified as “strongly associated” with the disorder.
According to the paper, reported by the SCMP, mutations in this gene were believed to contribute to developmental deficits, speech difficulties, repetitive behaviours, and epilepsy.
Li Dali, a professor of Life Sciences at East China Normal University, who is not an author of the paper, said “This was the first effective treatment of mice with ASD-related mutations using base editing in the brain, according to Shenzhen-based autism media platform Dami & Xiaomi”.
“The treatment successfully restored MEF2C protein levels in several brain regions and reversed the behavioural abnormalities in MEF2C-mutant mice,” the paper said.
However, for some patients, the disorder may not be caused by a single nucleotide variation like it was for the mice in the study, but rather by more complex mutations that would be difficult to edit.
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