Splice shifting oligonucleotides for use in the treatment of diseases characterized by altered inclusion of microexons

Lourdes Ruíz Desviat (Opfinder), Ainhoa Martínez Pizarro (Opfinder), José Javier Lucas Lozano (Opfinder), Sara Picó Del Pino (Opfinder), Brage S Andresen (Opfinder)

Publikation: Patent

Abstract

The present invention refers to splice shifting oligonucleotides (SSOs), targeting the downstream 3' splice acceptor site relative to microexons, which are mis-spliced in various diseases, particularly in neurological diseases like autism spectrum disorders (ASD) and schizophrenia.

OriginalsprogEngelsk
IPCC12N15/113
PatentnummerWO2023057575
Land/OmrådeDanmark
Prioritetsdato07/10/2021
PrioritetsnummerEP20210382898
StatusUdgivet - 13. apr. 2023

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