首页>
外国专利>
NUCLEIC ACID CONSTRUCTIONS AND GENETIC RETAIL VECTORS FOR USE IN THE TREATMENT OF WILSON'S DISEASE AND OTHER DISEASES
NUCLEIC ACID CONSTRUCTIONS AND GENETIC RETAIL VECTORS FOR USE IN THE TREATMENT OF WILSON'S DISEASE AND OTHER DISEASES
展开▼
机译:核酸构建和遗传零售载体,用于治疗威尔逊氏病和其他疾病
展开▼
页面导航
摘要
著录项
相似文献
摘要
The invention relates to a nucleic acid construct carrying ATP7B protein, an expression vector and a viral particle comprising the nucleic acid construct, and their use for treatment of Wilson's disease and other conditions caused by a deficiency or dysfunction of ATP7B protein. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.
展开▼