| 摘要 |
The present invention is directed to a method for providing AAV mediated gene therapy to a patient, comprising administering to a patient a replication-defective adeno-associated virus particle which infects a cell in the patient, the particle having therein a gene encoding a protein needed by the patient, the gene being operatively linked for expression in the cell, and at about the time of above-administering step, also administering to the patient an immunosuppressant that suppresses the patient's humoral immune response. The present invention is also directed to pharmaceutical compositions comprising the above described adeno-associated virus and humoral immuno-suppressant in a pharmaceutically acceptable carrier. Examples of proteins expressed by the above-described vectors include erythropoietin, thrombopoietin, human growth factor, leptin, Factor VIII, Factor IX, Factor Xa and the like. |
