jennin64

Each of these two participants received a short course ofglucocorticoid therapy, which rapidly normalized the liver enzymes andmaintained FIX levels in the range of 3–11% of normal values.

這兩個參與者都接受短期的糖(腎上腺)皮質激素療程，這可快速正常化肝酵素並將凝血因子含量維持在正常值的3-11%的範圍內。

No immune response to the FIX transgene product or products of alternateopen-reading frame translation could be detected.

並未偵測出對凝血因子轉殖產物或可變開放編閱轉譯產物的免疫反應。

These immunological findings are in agreement with previous results ofcapsid-specific T-cell responses in a Phase I/II study of AAV2–hFIX genetransfer.

這些免疫學上的發現跟先前AAV2–hFIX基因轉移一、二階段研究中衣殼蛋白特異性T細胞的結果相一致。

These data suggest that, for liver-directed gene transfer, there may be athreshold vector dose at which detection of capsid T-cell responses are clinicallyrelevant to the outcome of gene transfer and maneuvers to optimally suppressthese immune responses should be identified.

這些資料提到，對於肝臟為導向的基因移轉，可能有個臨界假體劑量，在這劑量下，衣殼蛋���T細胞的偵測在臨床上與基因移轉的結果有所關聯，而抑制這些免疫反應的最佳手法得以被鑑別出來。

This study has reopened and has recruited four more patients, and there areplans to continue a modified trial that will be open for further recruitment inlate spring 2013.

本研究重啟並多徵募了四位病患，在2013年晚春還有計畫進一步開放募集接續進行改良過的試驗。

A new Phase I/II trial has been approved to begin testing the safety, efficacyand optimal dose of an AAV8 vector containing the FIX Padua (R338L) constructto be given intravenously, and is now open to recruitment.

一項一、二階段試驗業經核准開始測試含有Padua (R338L)型凝血因子結構之靜脈注射腺相關病毒載體8型的安全性、功效跟最佳劑量，此刻正開放招募中。