April 07, 2025

JNCASR's new autism therapy to help patients become more self-sufficient

Scientists at JNCASR have made a remarkable breakthrough in autism treatment by targeting specific gene modifications in the brain. Their research focuses on a novel therapy that can potentially restore neuronal function and improve learning capabilities in patients with autism spectrum disorder. By using a specialized nanosphere-based approach, the team successfully demonstrated improved brain protein acetylation in mice models. This groundbreaking study offers hope for developing more effective interventions that could significantly enhance the quality of life for individuals with autism.

"This opens a very optimistic door for ASD therapy" - JNCASR Research Team

Researchers from Jawaharlal Nehru Centre for Advanced Scientific Research (JNCASR), an autonomous institute of the Department of Science and Technology (DST) have developed a new therapy that may help patients suffering from autism or intellectual disability to lead a life less dependent on others.

Key Points

1 Novel gene therapy targets brain protein acetylation in autism

2 Researcher Tapas K Kundu discovers innovative treatment approach

3 Nanosphere-based intervention shows promise in restoring neuronal function

4 Potential to improve patient independence and learning capabilities

Current therapeutics prescribed to treat autism spectrum disorder (ASD) or intellectual disability are mostly related to alleviating the symptoms. These do not correct the phenotypes observed in neurodevelopmental disorders, especially after brain development.

In a study on mice, the team led by Tapas K Kundu and James Clement from JNCASR, found that a gene that gets repressed in the brain of patients with autism.

“In mice with mutated syngap gene -- which resembles humans with mutated syngap gene (present in autistic patients) -- the acetylation of DNA-associated proteins, histones, or proteins that provide structural support for chromosomes is repressed in the brain,” said the team. The epigenetic enzyme behind this acetylation seems to be KAT3B or p300, they said.

Kundu’s group had previously discovered an activator of this enzyme, TTK21.

Upon conjugating this activator with glucose-derived nanosphere (CSP-TTK21) and feeding to the Syngap1 autistic mice, the researchers could induce acetylation in the brain.

In the research, published recently in the journal Aging Cell, the CSP-TTK21 was found to restore neuronal function, learning, and memory, and induce neuronal rearrangements in Syngap1 mice, mainly when administered after the brain is considered to be developed (adolescents in human beings).

This report not only directly connects histone acetylation with autism, for the first time, but also opens a very optimistic door for ASD therapy, said the researchers.

The study provides a new potential therapeutic option by targeting epigenetic modifications in Syngap1-related intellectual disability /ASD that can restore the deficits to an extent that will enable the patient to lead a life less dependent on others.

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