RNA Interference Therapeutics and Human Diseases

RNA Interference Therapeutics and Human Diseases

Copyright: © 2018 |Pages: 14
DOI: 10.4018/978-1-5225-2255-3.ch042
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Defective protein synthesis leads to diseases. If protein synthesis can be controlled, disease causing molecules can be tailored in some way. This is the perception behind RNA interference. RNA interference (RNAi) Therapeutics is branch of medicine which deals with the treatment of diseases while controlling the gene expression at RNA level. The motive of this chapter is to discover the state- of-art of RNAi Therapeutics, to explore various techniques used by RNAi Therapeutics to fight from diseases and discuss the future prospects of it.
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Cells are the building blocks of life. Inside nucleus of a cell, DNA and RNA molecules reside. RiboNucleicAcid (RNA) molecule is a single stranded sequence consisting of four bases Adenine (A), Guanine (G), Cytosine (C) and Uracil (U). This single stranded sequence is known as the primary structure of RNA (Tinoco & Bustamante,1999). The backbone of RNA contains ribose as sugar. Phosphate groups are also attached to ribose. RNA can be synthesized from DNA. DNA is another molecule which is double stranded molecule. DNA consists of nucleotides that contain a phosphate group, a sugar group and a nitrogen base. Nitrogen bases are Adenine (A), Guanine (G), Cytosine (C) and Thymine (T).

Key Terms in this Chapter

siRNA: Small interfering RNA is also known as silencing RNA or short interfering RNA. siRNA is formed from double-stranded RNA molecular sequence of length 20-25 base pairs and is used for knockdown of long noncoding RNAs genes.

RNAi Therapeutics: Altering gene coding at mRNA level is called RNAi Therapeutics. RNA Therapeutics drug are designed to increase or decrease the production of a protein involved in a disease.

RNA Interference: The mechanism of RNA Interference (RNAi) involves treating the disease causing gene with specific small interfering RNA (siRNA) that targets the specific messenger RNA (mRNA).

Gene Silencing: The process of prevention of gene expression process during translation or transcription process is called gene silencing.

LUNAR Technology: LUNAR stands for Lipid-enabled and Unlocked Nucleic Acid modified RNA delivery technology. In this technique, lipid particle is fused into the cell membrane that leads to biodegradation process and delivers the required RNA.

PMO: PMO (phosphorodiamidate morpholino oligomer) are synthetic RNA who retards the gene expression in order to prevent cells from making a targeted protein. They are molecular structures that are binded to RNA at specific position.

Gene Expression: It is the process of formation of protein, intermediate steps being conversion from DNA to mRNA.

dsRNA: dsRNA stands for double stranded RNA. Sence and antisence RNA together form dsRNA.

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