Galectin-1: an innovative therapeutic approach in dysferlinopathies

American researchers have developed a therapy aimed at correcting the phenotypes observed in dysferlinopathies (Miyoshi-type distal myopathy and LGMD type R2). Galectin-1, a beta-like galactoside binding protein, was genetically engineered and injected into model mice: 

  • galectin-1 is thought to play a role in membrane repair and inflammation, two mechanisms involved in dysferlinopathies; 
  • in treated mice, markers of inflammation and those of membrane fusion are effectively improved;
  • studies on human cells deficient in dysferlin have produced similar results.

These data constitute proof of concept for future therapeutic trials in humans.


Therapeutic Benefit of Galectin-1: Beyond Membrane Repair, a Multifaceted Approach to LGMD2B. Vallecillo-Zúniga ML, Poulson PD, Luddington JS et al. Cells. 2021 Nov 17;10(11):3210.