New hope for rare genetic condition

Embargoed until: Publicly released: 2025-07-02 15:05

Australia; International; VIC

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WEHI researchers have discovered an important role of an enzyme called Microrchidia CW-type zinc finger 2 (MORC2), which modifies the structure of DNA for gene expression. Mutations in MORC2 can lead to Charcot-Marie-Tooth disease, a severe genetic condition that causes progressive muscle weakness and loss of sensation. Understanding how MORC2 functions opens the door to developing new therapies. By targeting its activity, researchers may be able to design drugs that restore its function and slow or stop disease progression.