Nucleic acid therapeutics

  1. DNA central dogma
  2. 2 types of nucleic acids therapeutics
    • nucleosides/nucleotides
    • oligonucleotides
  3. types of oligonucleotides
    • recombinant DNA
    • anti gene
    • antisense
    • decoy
    • aptamer
    • NAzymes
    • RNAi
  4. purpose of nucleoside/nucleotide drugs
    they are incorporated in the DNA/RNA chain by polymerases and stops transcription/translation
  5. What is gene therapy?
    treating genetic disease using normal and functional genes usually a gene with normal functionality is introduced into a diseased cell. The new gene expressed the correct protein for normal functions
  6. what does the antigene approach do?
    a single stranded DNA sequence forms a triplex wight he double stranded DNA---> this inhibits the replication and transcription abilities of the cell
  7. what does the decoy approach do?
    short DNA duplex that binds to transcription factors and inhibit transcription
  8. what does the antisense approach do?
    short single stranded DNA/RNA sequence that specifically binds to its complementary counterpart in the gene---> this inhibits translation
  9. what does the NAzyme approach do?
    single stranded DNA or RNA moles catalyzes cleavage of chemical reaction ---->inhibit protein synthesis
  10. what does the aptamer approach do?
    single stranded DNA/RNA molecules with bind properties that act as agonists and antagonists to modulate protein functions by binding
  11. what does RNAi do?
    they are double stranded RNA sequences of 70 nucleotides (hairpin RNAs) that are expiated to the cytoplasm where dicer enzymes cleaves them into small interfering RNAs about 21 nucleotides long. The siRNAs antisense strand then incorporates into an RNA induced silencing complex which attach to and degrade its complementary target messenger RNA, reducing or stopping the expression of proteins.
  12. what are the main problems with nucleic acid drugs
    • 1. rapid degradation by nucleases
    • 2. biocompatibility
    • 3.ethical
    • 4. delivery into the nucleus
  13. what is a possible way to increase stability of nucleic acid drugs?
    induce chemical modifications to backbone, base, sugar and terminal end of nucleotide to improve resistance to nuclease, help with permeability, improve target specificity
Card Set
Nucleic acid therapeutics