What are oligonucleotide drugs?

Human Anatomy & Physiology (11th Edition)
11th Edition
ISBN:9780134580999
Author:Elaine N. Marieb, Katja N. Hoehn
Publisher:Elaine N. Marieb, Katja N. Hoehn
Chapter1: The Human Body: An Orientation
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Problem 1RQ: The correct sequence of levels forming the structural hierarchy is A. (a) organ, organ system,...
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What are oligonucleotide drugs?

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oligonucleotide drugs or Antisense oligonucleotides are are a short stretch of single stranded DNA or RNA that is often complementary to an infectious mRNA or disease mRNA that is made during the time of infirmity.

these antisense molecules hybridise with the target mRNA and prevent translation by degrading the antisense oligo-target mRNA hybrid by RNase H.

chemically-modified antisense oligonucleotides are often used in the therapy and they are taken up the cell by receptor-mediated endocytosis.

Antisense oligonucleotides are currently used in many life threatening diseases like

  1. cancers
  2. Batten disease
  3. CMV retinitis
  4. Duchenne muscular dystrophy
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