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Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice

Marcela P Cataldi1, Peijuan Lu1, Anthony Blaeser1

  • 1McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Carolinas Healthcare System, Charlotte, NC, 28203, USA.

Nature Communications
|August 29, 2018
PubMed

Insights

Ribitol, a sugar alcohol, partially restores functional O-mannosylation of alpha-dystroglycan (α-DG) in a muscular dystrophy model. This experimental therapy improves muscle pathology and function in FKRP mutant mice.

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Genetics

Background:

  • O-mannosylated α-dystroglycan (α-DG) is crucial for cell adhesion and signaling.
  • Defects in α-DG glycosylation are linked to cancer progression and muscular dystrophies.

Purpose of the Study:

  • To investigate the therapeutic potential of ribitol in restoring functional α-DG (F-α-DG) glycosylation.
  • To evaluate ribitol's efficacy in a mouse model of FKRP-related congenital muscular dystrophy.

Main Methods:

  • Utilized a mouse model with a P448L mutation in the FKRP gene.
  • Administered ribitol orally to assess its impact on ribitol-5-phosphate and CDP-ribitol levels.
  • Evaluated F-α-DG levels in skeletal and cardiac muscles.
  • Assessed muscle pathology, cardiac fibrosis, and functional improvements.

Main Results:

  • Ribitol administration restored therapeutic levels of F-α-DG in skeletal and cardiac muscles.
  • Treatment reduced skeletal muscle pathology and cardiac fibrosis.
  • Ribitol improved skeletal and respiratory functions in FKRP mutant mice.

Conclusions:

  • Ribitol shows promise as a novel therapeutic agent for FKRP-related muscular dystrophy.
  • Oral ribitol administration is a low-risk, easily administered therapy.
  • Restoring F-α-DG levels via ribitol offers a new treatment strategy for dystroglycanopathies.

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