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.
April 07, 2025
JNCASR's new autism therapy to help patients become more self-sufficient
"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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