de Feyter R, Li P
Gene Shears Pty Ltd, GPO Box 1600, Canberra, ACT 2601, Australia.
Curr Opin Mol Ther. 2000 Jun;2(3):332-5.
Ribozymes (catalytic RNAs) can be made to specifically cleave target RNAs that are involved in disease conditions and therefore have potential as therapeutic agents. Gene Shears Pty Ltd is developing hammerhead ribozyme technology for therapy against HIV infection, targeting either the tat gene or the RNA packaging sequence (Psi) of HIV. These ribozymes have been expressed from constructs that were introduced into hematopoietic cells in culture, thereby protecting the cells against viral infection. Two phase I clinical trials are underway to test the safety and feasibility of the approach with the anti-tat ribozyme in human subjects.
核酶（催化性RNA）能够被制备成特异性切割与疾病状态相关的靶RNA，因此具有作为治疗剂的潜力。基因剪切私人有限公司正在研发锤头状核酶技术用于治疗HIV感染，其靶向HIV的tat基因或RNA包装序列（Ψ）。这些核酶已从导入培养的造血细胞中的构建体表达，从而保护细胞免受病毒感染。两项I期临床试验正在进行，以测试在人类受试者中使用抗tat核酶这种方法的安全性和可行性。
