Groundbreaking Development: University of Tokyo Researchers Announce Successful Creation of Fragile X Syndrome Marmoset Model,東京大学


Groundbreaking Development: University of Tokyo Researchers Announce Successful Creation of Fragile X Syndrome Marmoset Model

Tokyo, Japan – September 4, 2025 – The University of Tokyo is proud to announce a significant scientific breakthrough: the successful development of a marmoset model for Fragile X syndrome. This achievement, detailed in a recent publication, marks a pivotal moment in the pursuit of understanding and treating this complex neurodevelopmental disorder.

Fragile X syndrome is a genetic condition that is the most common inherited cause of intellectual disability and autism spectrum disorder. It arises from a mutation in the FMR1 gene, leading to a deficiency in the fragile X mental retardation protein (FMRP), which is crucial for normal brain development. Despite extensive research, effective treatments remain elusive, largely due to the challenges in developing suitable animal models that accurately replicate the multifaceted symptoms of the human condition.

The research team at the University of Tokyo has overcome these hurdles by successfully engineering marmosets to carry a mutation in their FMR1 gene, mirroring the genetic cause of Fragile X syndrome in humans. Marmosets, a type of small New World monkey, offer several advantages as research models. Their genetic makeup and brain structure share significant similarities with humans, and their relatively short gestation period and high reproductive rates facilitate more efficient research. Crucially, their social behaviors and cognitive abilities provide a platform for investigating the complex behavioral and neurological deficits associated with Fragile X syndrome.

This newly developed marmoset model is expected to offer unprecedented opportunities for researchers to:

  • Elucidate Disease Mechanisms: The model will enable a deeper understanding of how the FMR1 gene mutation impacts brain development, neuronal function, and synaptic plasticity at various stages of life.
  • Test Novel Therapies: Researchers can now rigorously evaluate the efficacy of potential therapeutic interventions, including gene therapy, pharmacological treatments, and behavioral interventions, in a preclinical setting that closely approximates human biology.
  • Investigate Behavioral Phenotypes: The model will be instrumental in studying the specific behavioral characteristics of Fragile X syndrome, such as cognitive impairments, social deficits, and sensory sensitivities, leading to the development of more targeted therapeutic strategies.
  • Advance Precision Medicine: By studying the precise genetic and molecular underpinnings of the syndrome in this model, researchers can pave the way for more personalized and effective treatments for individuals affected by Fragile X syndrome.

The successful creation of this marmoset model represents a testament to the dedication and innovative spirit of the University of Tokyo’s research community. This advancement holds immense promise for accelerating the development of effective treatments and ultimately improving the lives of individuals and families affected by Fragile X syndrome worldwide. The University of Tokyo is committed to continued collaboration and advancement in this critical area of medical research.


脆弱X症候群のマーモセットモデル開発に成功


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東京大学 published ‘脆弱X症候群のマーモセットモデル開発に成功’ at 2025-09-04 15:00. Please write a detailed article about this news in a polite tone with relevant information. Please reply in English with the article only.

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