Brain model shows promise in treating FCMD

By The Science Advisory Board staff writers

October 1, 2021 -- A model using stem cell-derived muscle and brain organoids for Fukuyama congenital muscular dystrophy (FCMD)-related brain effects gives new hope for treatment, according to research published in the October 22 issue of iScience.

A team led by Mariko Taniguchi-Ikeda from the Department of Clinical Genetics of Fujita Health University in Toyoake, Aichi, Japan, touted the effectiveness of a small compound called Mannan-007 (Mn007) in reducing FCMD-related defects.

Researchers transformed pluripotent stem cells derived from a patient with FCMD into skeletal muscle and brain organoids, which are artificially grown miniature organs resembling the brain.

They found that Mn007 could restore the levels of alpha-dystroglycan, a protein required for maintaining muscle integrity, in both the muscle and brain models tested. Brain organoids treated with Mn007 also showed partial improvements in radial glial architecture and neuronal migration, resulting in fewer development defects. (iScience, October 22, 2021, Vol. 24:10, 103140.)

The team said while a cure for FCMD could still be years away, their findings are "an important landmark" in tackling such diseases.

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