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A decoy-based gene therapy targeting CUGexp-DMPK transcripts in DM1

Archive ouverte | Matloka, Magdalena | CCSD

International audience. Deleterious interactions of RNA binding factors with expanded CUG repeats leads to functional loss of MBNL1 resulting in alternative splicing misregulations and ultimately, to DM1 symptoms. C...

A Decoy-Based Gene Therapy to Inhibit RNA Toxicity Associated with Expanded CUG

Archive ouverte | Arandel, Ludovic | CCSD

International audience. "Introduction: Nuclear-retained CUGexp-DMPK transcripts sequester MBNL splicing factor hampering its alternative splicing regulatory function. Here, we develop a novel strategy using an engin...

Decoy gene therapy to reverse RNA toxicity in DM1

Archive ouverte | Arandel, Ludovic | CCSD

International audience. "Introduction: Currently, a number of RNA-based therapeutic strategies for DM1 are beingdeveloped, including small molecule and antisense oligonucleotide approches. Here weassessed a gene the...

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