Discover and read the best of Twitter Threads about #AAV

Most recents (9)

🧵 $EH Lets talk about Ehang and the secret ace up their sleeve.

🧠 Let us talk about the brain of future UAM operators - the Command and Control (C&C) center of Ehang.

#UAM #eVTOL #AAV #UAV #drones #SmartCity #EH216
@aktieuniverset
1/
This key piece of software is responsible for keeping track of your autonomous drone fleet to ensure safety and efficiency. It has 5 main purposes:

🖥 Monitoring
🛫 Dispatching
🛂 Controlling
⚠️ Warning
🧠 Centralizing

Lets go through each area in depth!
2/
🖥 Monitoring
The holistic overview of your entire fleet, regardless of product type:

- eVTOL
- UAV
- AAV

This part of the system allows you to see where every unit is and the direction they are moving as well as their current flight route.
3/
Read 25 tweets
1/This July’s @aspneph pathology webinar was all about pediatric Antineutrophil Cytoplasmic Antibody (ANCA)- associated vasculitis (AAV). Let's kick off this pediatric AAV tweetorial✍️with a quick question #NephTwitter
🤔 What is the role of ANCA antibodies❓
2/
✅Diagnostic marker and Pathogenic in nature
▶️ Proteinase-3 (PR3) and Myeloperoxidase (MPO) antigens are sequestered in neutrophil primary granules
▶️ Antigen exposure triggers immune response leading to endothelial activation
linkinghub.elsevier.com/retrieve/pii/S…
3/
Mechanisms
📌Defective neutrophil apoptosis, leading to NETosis
📌Inefficient clearance of PR3/MPO
📌Antimicrobial antibodies cross reacting with PR3/MPO
📌Medication induced
Read 21 tweets
Can directed evolution of the #AAV capsid yield useful lung gene therapy vectors? My paper with @VivianGradinaru at @Caltech, out today in @MolTherapy – Methods and Clinical Development (cell.com/molecular-ther…), aims to answer this question. 1/8
Compared to the brain, liver, muscle, eye, or inner ear, developing specialized AAV variants for the lung has received much less focus. This is despite the prevalence of lung-specific genetic disorders (i.e. Idiopathic Pulmonary Fibrosis). 2/8
Our lab specializes in developing gene therapy vectors for the brain, but the disparity in gene therapy tools for lung compared to other organs inspired us to see if the methods we use in the brain could be applied to this problem. 3/8
Read 8 tweets
@chowdarypm, @ProfMakris, and others report another successful #genetherapy for #hemophilia B clinical trial using the hyperactive FIX variant Padua.
tinyurl.com/346ty35v

🧵 on the clinical development of FIX Padua in gene therapy:

1/7
This is now the 4th successful Phase1/2 #genetherapy study using #AAV vectors using FIX Padua. The initial demonstration of the benefit of FIX Padua in #genetherapy for #hemophilia B was in a study led by @LindseyAGeorge.

nejm.org/doi/full/10.10…

2/7
Read 7 tweets
#SciTwitter After a lot of research and asking around, I'm making the lab equipment recommendations 🧵 I wish I'd had 2 months ago. RT/share with a #newPI or startup 🔬⚗️🛒
Note, much of the equipment hasn't arrived yet, will add comments after actual use.
-80 #freezer
Two clear winners: PHC (@panasonic) and Stirling Ultracold. Both low energy, quiet, reliable. We went with PHC because I know those to last many years, and slightly cheaper.
Thanks @MarcoJost_ @letUbeU @aryelipman #MBCBiolabs
-20 #freezer
Less clear, many viable options. We ended up getting a split of PHC MDF -30 (recommended as quieter) and much cheaper Corepoint Scientific/@VWR, will see which we prefer. Thermo hasn't failed #MBCbiolabs, but $$$ and several people said poor customer support.
Read 14 tweets
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 🐭 & ♂️♀️
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.
Plus a casual four new crystal structures, for #AAV capsids produced either in Sf9 cells or HEK293.
Read 3 tweets
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...
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.
Read 3 tweets
[Thread]
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?
Read 8 tweets
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. Image
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!
Coming up in 10 minutes! in session "Aav vectors preclinical and proof of concept studies in optimizing the toolbox". #ASGCT20
Read 3 tweets

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