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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
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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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