uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma in Hemophilia B Gene Therapy Program
uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma in Hemophilia B Gene Therapy Program
Multiple analyses conducted by an independent laboratory and reviewed by leading external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample. The integration events were distributed randomly across the genome, and there was no evidence of clonal expansion or any dominant integration event. Additionally, whole genome sequencing of the tumor confirmed that the patient had several genetic mutations that are characteristic of HCC and are independent of vector integration. Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.
Multiple analyses conducted by an independent laboratory and reviewed by leading external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample. The integration events were distributed randomly across the genome, and there was no evidence of clonal expansion or any dominant integration event. Additionally, whole genome sequencing of the tumor confirmed that the patient had several genetic mutations that are characteristic of HCC and are independent of vector integration. Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.
由獨立實驗室進行並經該領域領先的外部專家審查的多項分析表明,患者組織樣本中的AAV載體整合極為罕見,佔樣本細胞的0.027%。整合事件在基因組中隨機分佈,沒有克隆擴張或任何顯性整合事件的證據。此外,腫瘤的全基因組測序證實,患者有幾個肝癌特有的、與載體整合無關的基因突變。最後,腫瘤和鄰近組織的基因表達分析表明,肝臟處於癌前狀態,這與該患者易患肝癌的幾個危險因素一致。
譯文內容由第三人軟體翻譯。
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