Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1

Irene González-Martínez; Estefanía Cerro-Herreros; Marc Carrascosa-Sàez; Andrea García-Rey; Diego Piqueras-Losilla; Anna Colom-Rodrigo; Nadjeda Moreno; Mouli Chakraborty; Aline Huguet-Lachon; Anchel González‐Barriga; Neia Naldaiz-Gastesi; Martxel Dehesa; N. Barquero; Nuria Barquero; Miguel A. Varela; Adolfo López de Munain; ramon eritja; Geneviève Gourdon; Arturo López Castel; M. Pérez-Alonso; Rubén Artero; Rubén Artero

doi:10.1016/j.ajhg.2026.01.016

Key Points

To explore the muscle uptake of chemically optimized miR-23b antisense oligonucleotides as potential treatments for myotonic dystrophy type 1.
Utilization of chemically optimized miR-23b antisense oligonucleotides
Assessment of muscle uptake in animal models
Comparative analysis of oligonucleotide effectiveness
Significantly enhanced muscle uptake of miR-23b oligonucleotides observed
Positive correlation between oligonucleotide optimization and muscle delivery
Potential therapeutic benefits indicated for myotonic dystrophy type 1 treatment

Implication

Experimental findings reveal improved muscle uptake of miR-23b oligonucleotides for myotonic dystrophy type 1, suggesting potential treatment benefits.

Discussion

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Cite This Study

González-Martínez et al. (2026) studied this question.

synapsesocial.com/papers/699a9d8e482488d673cd370b https://doi.org/10.1016/j.ajhg.2026.01.016

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Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1

Key Points

Implication

Discussion

Cite This Study

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1

Key Points

Implication

Discussion

Cite This Study

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