COMPOUNDS FOR USE IN THE TREATMENT OF DISEASES BASED ON THE EXPRESSION OF TOXIC TRANSCRIPTS

The invention relates to compounds for use in the treatment of diseases based on the expression of toxic transcripts. The invention relates to peptide molecules, specifically hexapeptides, for the prevention and/or treatment of diseases having an etiology based on the presence of toxic transcripts c...

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Bibliographische Detailangaben
Hauptverfasser: ARTERO ALLEPUZ, RUBEN DARIO, ORZAEZ CALATAYUD, MARIA DEL MAR, LLAMUSI TROISI, MARIA BEATRIZ, GARCIA LOPEZ, AMPARO, PEREZ PAYA, ENRIQUE, PEREZ ALONSO, MANUEL
Format: Patent
Sprache:eng ; spa
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Zusammenfassung:The invention relates to compounds for use in the treatment of diseases based on the expression of toxic transcripts. The invention relates to peptide molecules, specifically hexapeptides, for the prevention and/or treatment of diseases having an etiology based on the presence of toxic transcripts comprising repeating units CUG, CCUG, CGG, CAG and AAG, preferably: DM1 SCA8, DM2, FXTAS, HD and FA. Compuestos para ser usados en el tratamiento de enfermedades basadas en la expresión de transcritos tóxicos. La presente invención hace referencia a moléculas peptídicas, concretamente hexapéptidos, para la prevención y/o tratamiento de enfermedades cuya etiología se basa en la presencia de transcritos tóxicos que comprenden repeticiones CUG, CCUG, CGG, CAG y AAG, preferentemente: DM1 SCA8, DM2, FXTAS, HD y FA.