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European Patent (TR Translation)

Durumların Ve Hastalıkların Tedavisine Yönelik Karşı Anlamlı Oligomerler

Invention Abstract

SCN1A genindeki alternatif splays olayları, üretken olmayan mRNA transkriptlerine yol açabilir ve bu da anormal protein ifadesine yol açabilir ve SCN1A genindeki alternatif splays olaylarını hedefleyebilen terapötik ajanlar, Dravet Sendromu hastalarında fonksiyonel proteinlerin ifade düzeyini modüle edebilir ve/veya anormal protein ifadesini inhibe edebilir. Bu tip terapötik ajanlar, SCN1A, SCN8A veya SCN5A protein eksikliğinden kaynaklanan bir durumu tedavi etmek için kullanılabilir.

IPC Classes

C12N15/11C07K14/705C12Q1/6883

Parties

Inventor

Zhou Han Isabel Aznarez

Rights Holder

Stoke Therapeutıcs Inc. 45 Wıggıns Avenue Bedford, Ma 01730 A.B.D.

Attorney

Erdem Kaya (Erdem Kaya Patent Ve Dan. A.Ş.)

This record is compiled from official TPMK patent bulletins and is provided for information. The abstract text is as published, in Turkish. The current legal status (opposition, assignment, invalidation) requires a full register review. Ask our experts for a comprehensive patent search.