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Updated: Jan 18, 2026

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Más allá de la parada: los TRID de oxadiazol restauran la expresión de la proteína LRBA en inmunodeficiencias

Ignazio Fiduccia1, Emanuele Vitale1, Riccardo Varrica1

  • 1Department of Biological, Chemical and Pharmaceutical Sciences and Technologies, University of Palermo, Viale delle Scienze Parco d'Orleans II Ed. 17, 90128 Palermo, Italy.

Molecular therapy. Nucleic acids
|January 16, 2026
PubMed
Resumen

Nuevos fármacos llamados fármacos inductores de lectura traslacional (TRID) basados en 1,2,4-oxadiazol muestran la promesa de tratar la deficiencia de LRBA. Estos compuestos restauran eficazmente la expresión de la proteína LRBA, ofreciendo alternativas después de la retirada de la ataluren.

Palabras clave:
Oligonucleótidos: Terapias y Aplicacionesmutaciones sin sentidooxadiazolesmedicina de precisióninmunodeficiencia primarialectura traslacional

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