Design of highly specific cytotoxins by using trans-splicing ribozymes
نویسندگان
چکیده
منابع مشابه
Design of highly specific cytotoxins by using trans-splicing ribozymes.
We have designed ribozymes based on a self-splicing group I intron that can trans-splice exon sequences into a chosen RNA target to create a functional chimeric mRNA and provide a highly specific trigger for gene expression. We have targeted ribozymes against the coat protein mRNA of a widespread plant pathogen, cucumber mosaic virus. The ribozymes were designed to trans-splice the coding seque...
متن کاملSelf-splicing and trans-splicing ribozymes
The Journal of Clinical Investigation | August 2003 | Volume 112 | Number 3 The splicing activity of Group I and Group II introns can be harnessed as a molecular tool that may potentially revise any gene of interest. Although hurdles remain, progress has been made in the development of these ribozymes for therapeutic application. While gene therapy is showing promise in the treatment of some di...
متن کاملTrans-splicing ribozymes for targeted gene delivery.
Ribozymes are potential tools for genetic manipulation, and various naturally occurring catalytic RNAs have been dissected and used as the basis for the design of new endoribonuclease activities. While such cleaving ribozymes may work well in vitro, they have not proved to be routinely effective in depleting living cells of the chosen target RNA. Recently, trans-splicing ribozymes have been emp...
متن کاملComputational prediction of efficient splice sites for trans-splicing ribozymes.
Group I introns have been engineered into trans-splicing ribozymes capable of replacing the 3'-terminal portion of an external mRNA with their own 3'-exon. Although this design makes trans-splicing ribozymes potentially useful for therapeutic application, their trans-splicing efficiency is usually too low for medical use. One factor that strongly influences trans-splicing efficiency is the posi...
متن کاملDesign and Experimental Evolution of trans-Splicing Group I Intron Ribozymes.
Group I intron ribozymes occur naturally as cis-splicing ribozymes, in the form of introns that do not require the spliceosome for their removal. Instead, they catalyze two consecutive trans-phosphorylation reactions to remove themselves from a primary transcript, and join the two flanking exons. Designed, trans-splicing variants of these ribozymes replace the 3'-portion of a substrate with the...
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ژورنال
عنوان ژورنال: Proceedings of the National Academy of Sciences
سال: 1999
ISSN: 0027-8424,1091-6490
DOI: 10.1073/pnas.96.7.3507