2.Antisense lecture

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Overview
Recent advances in recombinant DNA technology have coincided with new insights into the control of cell growth and have highlighted nucleic acid therapy as an attractive alternative therapeutic approach to conventional drug intervention.
Nucleic Acid therapy is not the same as Gene Therapy
Gene therapy is the transfer of a functional whole gene into human cells to replace a mutated, disfunctional one.
Nucleic Acid Therapy can be described as the use of small oligonucleotides which recognise and bind to disease related molecules, and block their activity.

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Liposomes
Encapsulation within liposomes enhances uptake of antisense drugs.
Liposomes can deliver low, non toxic levels of oligonucleotide
Liposomes can be conjugated with a cell-specific binding site to direct delivery of the antisense oligonucleotiode to a specific tissue

After the oligo has been delivered:
Formation of the mRNA/AS DNA hybrid induces endogenous RNAse H which cleaves the mRNA component of the hybrid thereby stalling gene expression.

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