Oligonucleotide conjugates as potential antisense drugs with improved uptake, biodistribution, targeted delivery, and mechanism of action

M Manoharan - Antisense and Nucleic Acid Drug Development, 2002 - liebertpub.com
Antisense and Nucleic Acid Drug Development, 2002liebertpub.com
This review summarizes the effect of conjugating small molecules and large
biomacromolecules to antisense oligonucleotides to improve their therapeutic potential. In
many cases, favorable changes in pharmacokinetic and pharmacodynamic properties were
observed. Opportunities exist to change the terminating mechanism of antisense action or to
enhance the RNase H mode of action via conjugate formation.
This review summarizes the effect of conjugating small molecules and large biomacromolecules to antisense oligonucleotides to improve their therapeutic potential. In many cases, favorable changes in pharmacokinetic and pharmacodynamic properties were observed. Opportunities exist to change the terminating mechanism of antisense action or to enhance the RNase H mode of action via conjugate formation.
Mary Ann Liebert