RNA Interference Therapeutics and Human Diseases

RNA Interference Therapeutics and Human Diseases

Dolly Sharma, Shailendra Singh, Trilok Chand
Copyright: © 2019 |Pages: 15
DOI: 10.4018/978-1-5225-7489-7.ch006
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Abstract

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-the-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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Background

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).

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