Gene Therapy - Nucleic acid pharmaceuticals In Healthcare

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Gene Therapy

	About Gene therapy
	What are the genes?
	How does gene therapy work?
	Types of gene therapy
	Viral gene delivery systems
	Non-viral gene delivery systems
	Pro-drug activation therapy
	Two-gene therapy
	Nucleic acid therapeutic agents
	Antisense RNA production
	Antisense Oligonucleotides
	Nucleic acid pharmaceuticals
	Ribozymes - as therapeutic agents
	Oligonucleotide correction of genetic
	History of events - gene therapy

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Gene Therapy Introduction

Nucleic acid pharmaceuticals

Administration of oligonucleotides that attaches to transcription or translation factor could prevent expression of target gene.
Nucleic acid molecules are capable of binding to proteins, and by devising an oligonucleotide that binds to protein that does not normally bind to nucleic acid molecule and as a consequence of this attachment, inhibit functioning of this protein.

This type of oligonucleotide is called ‘Aptamer’. Potential anti-thrombin aptamer inhibits thrombin.

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