AMINO ACID COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS

• Main Authors: VIDYASAGAR SADASIVAN, XU XIAODONG, PRABHAKARAN SREEKALA, GROSCHE ASTRID, LIN SHANSHAN
• Format: Patent
• Language: Chinese; English
• Published: 23.07.2021
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

Provided herein are amino acid compositions useful for increasing the translocation of the cystic fibrosis transmembrane conductance (CFTR) protein from the cytoplasm to the plasma membrane, particularly in epithelial cells. Methods for increasing the concentration of CFTR in the plasma membrane, increasing chloride ion transport, and increasing water transport are also provided. These compositions and methods are useful in treating cystic fibrosis in subjects bearing one or more mutations in the CFTR protein. Use of these compositions for the treatment of cystic fibrosis and in the preparation of a medicament for the treatment of cystic fibrosis are also encompassed herein. 本文提供了可用于增加囊性纤维化跨膜电导(CFTR)蛋白从特别是在上皮细胞中的细胞质易位到质膜的氨基酸组合物。还提供了用于增加CFTR在质膜中的浓度、增加氯离子转运和增加水转运的方法。这些组合物和方法可用于治疗在所述CFTR蛋白中携带一个或多个突变的受试者的囊性纤维化。这些组合物在治疗囊性纤维化和在制备用于治疗囊性纤维化的药剂中的用途也涵盖在本文中。