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针对bcr-abl转录本的小干扰RNA在一名伊马替尼耐药慢性髓性白血病患者中的治疗应用。

Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia.

作者信息

Koldehoff M, Steckel N K, Beelen D W, Elmaagacli A H

机构信息

Department of Bone Marrow Transplantation, University of Duisburg-Essen, Hufelandstr. 55, 45122 Essen, Germany.

出版信息

Clin Exp Med. 2007 Jun;7(2):47-55. doi: 10.1007/s10238-007-0125-z. Epub 2007 Jul 4.

Abstract

RNA interference is referred to as the recently discovered process of sequence-specific, post-transcriptional gene silencing that is initiated by double-stranded RNA molecules known as small interfering RNAs (siRNA). We herein present a first report on the in vivo application of targeted non-virally delivered synthetic bcr-abl siRNA in a female patient with recurrent Philadelphia chromosome-positive chronic myeloid leukaemia (CML) resistant to imatinib (Y253F mutation) and chemotherapy after allogeneic haematopoietic stem cell transplantation. We found a remarkable inhibition of the overexpressed bcr-abl oncogene resulting in increased apoptosis of CML cells. In vivo siRNA application was well tolerated without any clinically adverse events. Our findings imply that the clinical application of synthetic siRNA is feasible, safe and has real potential for genetic-based therapies using synthetic non-viral carriers.

摘要

RNA干扰是指最近发现的一种序列特异性、转录后基因沉默过程,该过程由被称为小干扰RNA(siRNA)的双链RNA分子引发。我们在此首次报告了靶向非病毒递送的合成bcr-abl siRNA在一名患有复发性费城染色体阳性慢性髓性白血病(CML)的女性患者中的体内应用,该患者对伊马替尼耐药(Y253F突变)且在异基因造血干细胞移植后对化疗耐药。我们发现过表达的bcr-abl癌基因受到显著抑制,导致CML细胞凋亡增加。体内应用siRNA耐受性良好,无任何临床不良事件。我们的研究结果表明,合成siRNA的临床应用是可行、安全的,并且在使用合成非病毒载体进行基于基因的治疗方面具有真正的潜力。

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