Kashani-Sabet Mohammed
Auerback Melanoma Research Laboratory, Cutaneous Oncology Program, UCSF Comprehensive Cancer Center, Department of Dermatology, University of California San Francisco, CA 94115, USA.
Expert Opin Biol Ther. 2004 Nov;4(11):1749-55. doi: 10.1517/14712598.4.11.1749.
Ribozymes are RNA molecules with the capacity to effect sequence-specific cleavage of other transcripts. Since their initial discovery, there has been considerable interest in the development of ribozymes and other RNA therapeutics for gene therapy, particularly in the realm of cancer. However, as with other gene therapy applications, the delivery of ribozyme-based therapeutics to the target tissues of interest has represented a significant obstacle to the maturation of this technology to the clinical arena. This review will discuss the progress made so far in the use of non-viral methods for the systemic delivery of ribozymes for cancer gene therapy.
核酶是一类能够对其他转录本进行序列特异性切割的RNA分子。自首次发现以来,人们对开发用于基因治疗的核酶及其他RNA疗法产生了浓厚兴趣,尤其是在癌症领域。然而,与其他基因治疗应用一样,将基于核酶的治疗药物递送至目标靶组织一直是该技术走向临床应用的重大障碍。本综述将讨论目前在使用非病毒方法进行核酶全身递送用于癌症基因治疗方面所取得的进展。