Therapeutic systems using nucleotide sequences are devised to treat human disorders.
Small, single strand nucleotide sequences
(oligonucleotides) could hybridize to a specific gene or mRNA and reduce transcription or translation, lowering the amount of protein synthesized.
An oligonucleotides designed to bind to a gene and block transcription is called antigen oligonucleotides and one that base pairs with specific mRNA is an antisense oligonucleotides.
Binding of oligonucleotides to transcription factor responsible for
expression of specific gene. Double stranded oligonucleotides that attach to DNA binding proteins could prevent activation of transcription of specific genes.
Synthetic DNA molecules that bind to proteins and prevent them from functioning can be created.
Ribozymes are natural RNA sequences that bind and cleave specific RNA molecules. These could be engineered to target an mRNA and decrease the amount of
proteon.