Most of the Oligonucleotide Therapeutics Society (OTS) 6th annual conference has discussed therapeutic siRNA and RNAi. I'll be "live blogging" :) or rather note-taking on the blog when cool talks come up. Anytime people start talking about these technologies, it is usually to knock down a particular protein (inhibition) which also lends nicely to aptamer related work.
Talk given by Claes Wahlestedt, MD, PhD, Scripps Research Institute talked about BACE1 beta-secretase. Known to be involved in Alzheimers disease, cleaves amyloid beta. Inactivation by siRNA reduces by BACE-1 and provides recovery in diseased mice. Mayank is currently working on APP, with current results.
He also talked about BDNF (Brain-derived neurotrophic factor) for neuronal growth and maturation, synaptic plasticity, Alzheimer's disease, etc. The expression is most active is the Ectx portion of the brain in diseased patients. Knockdown of the antisense mRNA results in outgrowth of hippocampal nerve cells due to increased BDNF production. So, this may not be a good target (since it appears BDNF is good for the body). Recent paper Filion et al, Cell, October 15, 2010.
Jiehua Zhou, PhD, Beckman Research Institute, City of Hope working in John Rossi's lab is looking at aptamer siRNA/dendrimer/aptamer conjugates for gp120 as a therapeutic for HIV. There are a few HIV targets that would also be interesting. Arshia thought about CCR5 previously, It sits with CD4. gp41 and other exterior targets on.
Michael Lahn from Lilly Research suggested Survivin, also called baculoviral inhibitor of apoptosis repeat-containing 5 or BIRC5, may be a good target for cancer therapy. By inhibiting survivin activation pathways apoptosis and decrease in tumour growth are promoted. Survivin is upregulated in most human cancers and not present in normal differentiated cells. Therefore, it makes an ideal diagnostic and therapeutic target. It is also commercially available from ProSpec.
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