用于治疗SERPINC1相关病症的方法和组合物

本发明涉及iRNA例如双链核糖核酸(dsRNA)、靶向Serpinc1基因的组合物和使用这样的iRNA例如dsRNA、组合物来抑制Serpinc1的表达和治疗具有Serpinc1相关疾病例如出血病症诸如血友病的受试者的方法。 The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhib...

Full description

Saved in:
Bibliographic Details
Format Patent
LanguageChinese
Published 11.07.2025
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
Online AccessGet full text

Cover

More Information
Summary:本发明涉及iRNA例如双链核糖核酸(dsRNA)、靶向Serpinc1基因的组合物和使用这样的iRNA例如dsRNA、组合物来抑制Serpinc1的表达和治疗具有Serpinc1相关疾病例如出血病症诸如血友病的受试者的方法。 The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and to treat subjects having a Serpinc1-associated disease, e.g., a bleeding disorder, such as a hemophilia.
Bibliography:Application Number: CN202510290024