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GENE THERAPY
THE STATE OF THE ART

 Dr. Abdel Aziz El Bayoumi
Professor of Genetics

Dr. Khalid Al Ali
Lecturer of Genetics

Department of Biological Sciences
University of Qatar, Doha


11.1.5.2 RNA Strategies

RNA gene therapy approaches using the RNA for viral regulatory proteins includes the ribozymes and antisense DNA / RNA. These RNA mimics a viral binding site for the regulatory protein and captures it so that it is not available for viral replication.

The antisense RNA encodes untranslated RNA molecules, that binds with the sense strand RNA’s transcribed from the HIV genome. The formation of antisense –sense duplexes blocks translation and stimulates degradation of the unstable RNA complex (Welch, 1996).

The ribozyme, which is a molecule of RNA that has enzymatic activity against specific target RNA sequences. Ribozyme binds to complementary sequences on target RNA transcript, which cleaves the substrate RNA in a catalytic manner. In the case of RNA viruses such as HIV, ribozyme can cleave incoming viral genomic RNA prior to integration and can disrupt mRNA translation and progeny RNA genomes that ready for packaging to produce new virions (Yu, et al 1993 and Welch1996).

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