发明公开
EP0933997A1 METHODS AND COMPOSITIONS FOR LIVER SPECIFIC DELIVERY OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS 失效
方法和组合物治疗性分子在肝脏特异性交付期重组AAV载体

METHODS AND COMPOSITIONS FOR LIVER SPECIFIC DELIVERY OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
摘要:
Provided are methods for selectively expressing therapeutic molecules, such as secretory proteins, antisense molecules and ribozymes, in the liver. The methods find use in treating hepatic diseases or conditions. The methods also find use in treating any disease or condition in which systemic administration of the therapeutic substance, for example, a secretory protein, is desired. The methods involve administering to a mammalian patient having a need for liver expression of a therapeutic molecule an AAV vector containing a therapeutically effective amount of the therapeutic molecule. Also provided are novel vectors employable in these methods.
信息查询
0/0