LNA Gapmers for Treatment of Muscular Dystrophy


Researchers at Children's National Hospital developed Locked Nucleic Acid Gapmers (LNA Gapmers) for the treatment for facioscapulohumeral muscular dystrophy (FSHD). Expression of the DUX4 gene leads to FSHD, and our LNA gapmers suppress the expression of DUX4 and alleviate the symptoms of FSHD in vivo.


LNA Gapmers are nucleic acid based therapeutic agents that hybridize to a target sequence of mRNA, leading to RNaseH-mediated degradation of the mRNA molecule. The LNA Gapmers designed here target the DUX4 gene, suppressing the expression of its mRNA. Aberrant expression of DUX4 is implicated as the major contributor to the cause of FSHD. There is currently no cure for FSHD, and this Gapmer technology would represent the first effective biological treatment. The LNA Gapmers designed here have the following advantages over other nucleic acid based therapy strategies:

  1. High affinity to the target DUX4 mRNA.
  2. Efficient delivery in vivo without a viral vector.
  3. Resistant to nucleolytic degradation.


  • A novel therapeutic strategy for FSHD

Stage of Development

  • In vivo proof of concept studies completed, showing improved muscle function in mouse model.

Intellectual Property Status

  • PCT Patent Application filed, “Gapmers and Methods of Using the Same for Treatment of Muscular Dystrophy.” Filed Sept 19, 2018

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