Modified Nucleotides and Challenges of Clinical Oligonucleotides

Lots of cool modified nucleotides going into siRNA and aptamer related works that completely affects binding properties, folding, etc. Aptamer has to go through amplification (enzymatic) so less, but that may be gotten around by AM Biotech.

Michael Flanagan, PhD, Sirna Therapeutics gave a great talk about the challenges of pharmacokinetic and in pharmacodynamics with siRNA, but it applies to any future use of the aptamers we may develop. The questions are dose/schedule. This relates to what happens when the nucleic acid hits the body. Kinetics refer to how long the oligonucleotide is in the body whereas dynamics is how it affects the body adversely when in (i.e. toxicity, immune response, etc) as a function of dose.

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