DESIGN AND SELECTION OF RIBOZYMES FOR HIV THERAPY

描述（改编自研究者摘要）：本研究的目的 目前的建议是开发用于治疗的催化RNA或核酶的用途。 工作将继续在锤头，一个良好的特点RNA切割 试剂，重点是了解为什么嵌入在 大RNA通常比短寡聚体慢得多。 此外，本发明还提供了一种方法， 提出了一种实验方法来获得靶的元素速率常数 为了更好地在细胞内结合和分裂锤头， 与体外和体内实验相关。 最后，最近的发现 将研究交联锤头是有效的RNA连接酶 目的是将其用作RNA修复试剂。 为了扩大 可用于治疗的核酶库，两种体外 提出了选择策略，旨在确定新的 作用于蛋白质的核酶。 一个选择将搜索一个 脱磷酸酶，另一个是肽酶。

DESCRIPTION (Adapted from the Investigator's Abstract): The aims of this proposal are to develop the use of catalytic RNAs or ribozymes for therapy. Work will continue on the hammerhead, a well-characterized RNA cleavage reagent, with a focus on understanding why cleavage of a target embedded in a large RNA is often much slower than short oligomers. In addition, experiments are proposed to obtain the elemental rate constants of target binding and cleavage for a hammerhead inside cells in order to better correlate in vitro and in vivo experiments. Finally, the recent discovery that a crosslinked hammerhead is an effective RNA ligase will be studied with the aim of using it as an RNA repair reagent. In an attempt to expand the repertoire of ribozymes that could be used in therapy, two in vitro selection strategies are proposed that are intended to identify novel ribozymes that act on proteins. One selection will search for a dephosphorylase, the other a peptidase.