Please use this identifier to cite or link to this item: http://scholarbank.nus.edu.sg/handle/10635/120355
Title: DESIGN OF MINIMAL DUMBBELL-SHAPED DNA VECTORS FOR CODING AND NON-CODING RNA EXPRESSION
Authors: JIANG XIAOOU
Keywords: gene therapy, non-viral minial DNA vectors
Issue Date: 16-Jan-2015
Citation: JIANG XIAOOU (2015-01-16). DESIGN OF MINIMAL DUMBBELL-SHAPED DNA VECTORS FOR CODING AND NON-CODING RNA EXPRESSION. ScholarBank@NUS Repository.
Abstract: A MAJOR BARRIER FOR USING NON-VIRAL VECTORS FOR GENE THERAPY IS THE SHORT DURATION OF TRANSGENE EXPRESSION IN POSTMITOTIC TISSUES. PREVIOUS STUDIES SHOWED TRANSGENE EXPRESSION FROM CONVENTIONAL PLASMID FELL TO SUB-THERAPEUTIC LEVEL SHORTLY AFTER DELIVERY EVEN THOUGH THE VECTOR DNA WAS RETAINED, SUGGESTING TRANSCRIPTION WAS SILENCED IN VIVO. EMERGING EVIDENCE INDICATES THAT PLASMID BACTERIAL BACKBONE SEQUENCE IS RESPONSIBLE FOR THE TRANSCRIPTIONAL REPRESSION AND THIS PROCESS IS INDEPENDENT OF CPG METHYLATION. DUMBBELL-SHAPED VECTORS CONSISTING SOLELY OF ESSENTIAL ELEMENTS FOR TRANSGENE EXPRESSION (PROMOTER, POLY(A) SIGNAL, CODING GENES, RNA STABILIZING SEQUENCES ETC.) HAVE BEEN DEVELOPED TO CIRCUMVENT THESE DRAWBACKS. WE HAVE DESIGNED DUMBBELL-SHAPED VECTORS FOR SMALL NON-CODING RNA AS WELL AS PROTEIN-CODING GENE EXPRESSION. SOME NOVEL MOLECULAR FEATURES WERE INVESTIGATED AND SHOWN TO EITHER IMPROVE THE BIOLOGICAL ACTIVITY OF THE VECTOR OR EASE THE PRODUCTION. THIS MAY FACILITATE THE DE
URI: http://scholarbank.nus.edu.sg/handle/10635/120355
Appears in Collections:Ph.D Theses (Open)

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