Nucleic acid therapeutics

DNA-->RNA-->Protein

• nucleosides/nucleotides
• oligonucleotides

• recombinant DNA
• anti gene
• antisense
• decoy
• aptamer
• NAzymes
• RNAi

they are incorporated in the DNA/RNA chain by polymerases and stops transcription/translation

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

a single stranded DNA sequence forms a triplex wight he double stranded DNA---> this inhibits the replication and transcription abilities of the cell

short DNA duplex that binds to transcription factors and inhibit transcription

short single stranded DNA/RNA sequence that specifically binds to its complementary counterpart in the gene---> this inhibits translation

single stranded DNA or RNA moles catalyzes cleavage of chemical reaction ---->inhibit protein synthesis

single stranded DNA/RNA molecules with bind properties that act as agonists and antagonists to modulate protein functions by binding

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.

• 1. rapid degradation by nucleases
• 2. biocompatibility
• 3.ethical
• 4. delivery into the nucleus

induce chemical modifications to backbone, base, sugar and terminal end of nucleotide to improve resistance to nuclease, help with permeability, improve target specificity