Opening New Paths of Research, Israeli Scientists Use Worms to Mimic Muscular Dystrophy

Jerusalem, 15 May, 2024 (TPS) -- Israeli scientists have pioneered an innovative platform for replicating human muscle diseases in worms, marking a significant leap forward in disease modeling and drug screening for patients suffering from muscular dystrophy.

Muscular dystrophy refers to a group of genetic disorders characterized by progressive weakness and loss of muscle mass. There is no cure and treatment focuses on managing symptoms with physical therapy, assistive devices, respiratory support, medicine, or orthopedic surgery.

While muscular dystrophy itself is not typically directly fatal, the progressive muscle weakness and loss of function can lead to complications affecting various organs and systems in the body, potentially resulting in premature death.

Researchers from Bar-Ilan University working in conjunction with Dr. Amir Dori of the Sheba Medical Center in Ramat Gan, devised a novel approach utilizing extracellular vesicles (EVs) from patients with muscular dystrophy, the most common muscular disease. EVs are small membrane-bound particles, bubble-like, that are released by cells and play a crucial role in intercellular communication and the transfer of biomolecules between cells.

The EVs, isolated directly from patient blood samples, were transferred to C. elegans worms, resulting in the development of muscle atrophy akin to the human disease. Unlike traditional genetic models, which necessitate alterations in the organism’s genes to simulate disease conditions, the research team’s method leverages extracellular vesicles to induce disease pathology in the one-millimeter-long worms.

“The idea of taking extracellular bubbles from patients with muscular dystrophy and transferring them to worms was such a crazy idea that we were surprised by the results,” said Professors Chaya Brody and Sivan Kornblit, in whose laboratories the research was conducted.

The study primarily focused on Duchenne and Becker muscular dystrophy, debilitating genetic diseases affecting skeletal and cardiac muscles, predominantly in males.

“This platform allows us to create disease models specific to each patient, enabling comprehensive research into the physiological and genetic underpinnings of the condition,” the researchers said, offering hope for developing targeted therapies and personalized medicine.

The findings were recently published in Disease Models & Mechanisms, a peer-reviewed journal.