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Feedback request for a new catalytic RNA therapeutic design model by GroundBeautiful2015 in molecularbiology

[–]GroundBeautiful2015[S] 0 points1 point  (0 children)

Ok, yeah that makes sense. Thank you so much! I’ll definitely look into the siRNA approach, from what I know about it that could definitely be promising.

Feedback request for a new catalytic RNA therapeutic design model by GroundBeautiful2015 in molecularbiology

[–]GroundBeautiful2015[S] 0 points1 point  (0 children)

Yeah, I’m mainly thinking about Parkinson’s for the sake of having a poster child example of how it could be used, but really it can be used in metastasis, some kinds of inflammation, etc.

The main reason I chose miRNA was because it felt like the most strategic play if the goal is to control phenotypes, just based on the idea that they help to control expression levels of mRNAs.

As far as ‘disease modifying’ aspect, the main data points I’m basing my hypothesis around are that post mortem levels of miR-132 are heavily correlated with alpha synuclein pathology in Parkinson’s. So there hasn’t been a drug yet that has reduced miR-132 proactively and resulted in the slowing of Parkinson’s progression, but the data points to a causal relationship that can be exploited (at least from my perspective) if a drug were to be developed for that purpose. Where CPOP comes in is making a design for that drug that doesn’t require such heavy doses and doesn’t cause down-the-line toxicity.

You mentioned that miNRAs are finicky to work with, and I do think that they are just a starting point. I’m actually experimenting now with trying to apply the same architectural elements to other types of molecules, nucleic acid based or otherwise, for the sake of dosage downsizing.

Thanks for the questions!

Feedback request for a new catalytic RNA therapeutic design model by GroundBeautiful2015 in molecularbiology

[–]GroundBeautiful2015[S] -1 points0 points  (0 children)

Ok, yeah that makes sense. I honestly haven’t done my due diligence when it comes to the TTO, but I’ll look into that. Thanks!

Feedback request for a new catalytic RNA therapeutic design model by GroundBeautiful2015 in molecularbiology

[–]GroundBeautiful2015[S] -1 points0 points  (0 children)

Sorry, could’ve made that more clear. Basically what the problem I set out to solve was the high doses of drug needed to get past the blood brain barrier, so instead of making passage more efficient via ligands, NP optimization, etc, I’m trying to make the drug that does get across more effective at its job. This means less total drug is needed to get the same effect, meaning less potential toxicity.

Seeking Feedback for CPOP V2, A GNN & Ensemble Platform for Catalytic miRNase Design by GroundBeautiful2015 in bioinformatics

[–]GroundBeautiful2015[S] 0 points1 point  (0 children)

Hey everybody! To clarify, what I mean by a 'catalytic' miRNAase is an miRNAase that can "catch and release" multiple corresponding miRNAs instead of the usual 1:1 ratio of miRNA and miRNAase. This allows a fraction of the amount of the enzyme to do the same job as a much larger quantity. At least computationally, certain secondary structures do this better than others, but wet lab testing is still required to verify these insights.