Discover and read the best of Twitter Threads about #ASGCT20
Most recents (4)
Color me biased, but @Nicole_Paulk's #ASGCT20 award talk was like 2-3 #AAVbiology papers in one. First obs that:
1) #AAV genomes are epigenetically modified
2) #AAV capsids have PTMs
3) Both differ for 293 vs Sf9 production
4) In vivo potency affected, incl. #humanized 🐭 & ♂️♀️
1) #AAV genomes are epigenetically modified
2) #AAV capsids have PTMs
3) Both differ for 293 vs Sf9 production
4) In vivo potency affected, incl. #humanized 🐭 & ♂️♀️
A mountain of new topics for @ASGCTherapy members to explore!
🚨She also briefly mentioned observations of insect host cell impurities, which could be a major issue for #ClinicalTrials, especially with high doses for systemic (incl. non-rare!) disease🚨 Needs to be explored.
🚨She also briefly mentioned observations of insect host cell impurities, which could be a major issue for #ClinicalTrials, especially with high doses for systemic (incl. non-rare!) disease🚨 Needs to be explored.
Plus a casual four new crystal structures, for #AAV capsids produced either in Sf9 cells or HEK293.
Some talks I liked at #ASGCT20 today:
Sean Wang from Cepko lab applied SABER #FISH to (nature.com/articles/s4159…) #AAV genomes, tracking individual particles w high time res. I wonder if pH sensitive probes would allow imaging of intracellular trafficking...
Sean Wang from Cepko lab applied SABER #FISH to (nature.com/articles/s4159…) #AAV genomes, tracking individual particles w high time res. I wonder if pH sensitive probes would allow imaging of intracellular trafficking...
Faith Conroy at #UMass targeting Htt with siRNAs, and used intentional mismatches to improve specificity for SNP gene product over WT.
Beautiful Outstanding Achievement talk by Mavis Agbandje-McKenna. Lots of #AAV structure, and work with @asokan_duke and others to map out functional #AAV surface host interactions. Also work on non-#primate #AAVs, which could evade human neutralizing antibodies.
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Two #ASGCT20 morning sessions focused on lung delivery. Cool work, but it seems to me that the big issues for clinical stage haven't really been addressed:
Two #ASGCT20 morning sessions focused on lung delivery. Cool work, but it seems to me that the big issues for clinical stage haven't really been addressed:
#AAV can target specific cell types in the lung, and multiple groups have rescued mouse models of genetic diseases. But both new and old lung-trophic capsids seem to hit T2 epithelial cells, which turn over and lose non-integrating transgenes.
Why does the virus end up in these cells? Are the T1 cells designed to reject microbes? What about fibroblasts and other cells, that do get transduced in some tissues?
Gordian is speaking at the (virtual) #asgct2020. Come hear how we do entire #HighThroughput #GeneTherapy screens for #ComplexDisease, all in a single animals. We'll show large animal data, and some basic #AAV biology we learned along the way. Tuesday 13:45 EST, Room 312. 
There's some tough competition in the #ASGCT20 program today, especially from the juicy #AAV vector biology session.
But if you're curious about how #GeneTherapy can be extended to non-monogenic diseases, I think we've got the Tuesday afternoon market cornered!
But if you're curious about how #GeneTherapy can be extended to non-monogenic diseases, I think we've got the Tuesday afternoon market cornered!
Coming up in 10 minutes! in session "Aav vectors preclinical and proof of concept studies in optimizing the toolbox". #ASGCT20
