Top Tweets for #BaseEditor
YOLT-101
Another single-infusion #BaseEditor therapy for Heterozygous Familial Hypercholesterolemia
From #YolTech Therapeutics 🇨🇳
0.6 mg/kg N=3
Being effective in 1 wk
Sustained 70% ⬇️plasma PCSk9 & 50% ⬇️ plasma LDL-C till 16-wk
medRxiv 2025
https://t.co/QsKaf8gjOh
A customized CRISPR-Cas9 #BaseEditor targeting the ACTA2 gene has shown promise in extending survival and reducing disease symptoms in mouse models of MSMDS, a rare pediatric disorder. @MassGenBrigham @natBME https://t.co/ilpt17Un0l https://t.co/l7D9BirnBb
🚨 Excited to share our latest preprint!
@Garnettlab @MatthewACoelho
We introduce BEstimate, a flexible, comprehensive tool for designing and interpreting CRISPR-based editing experiments.
#CRISPR #BEstimate #BaseEditor
BEstimate: a computational tool for the design and interpretation of CRISPR base editing experiments https://t.co/9jMkwAxN31 #biorxiv_bioinfo
Design & Deliver #CRISPR #BaseEditor #PrimeEditor to your adult👤stem cell-derived (colon/ endometrium/hepatocyte) #Organoids
Plasmids
Electroporation
FACS + PiggyBac selection
Learn "Picking the right genome editing tool in 30 minutes!"😄(Fig 1 Table 1)
@CelottiMartina @TheCleversLab @geurts_maarten @STARProtocols 2024
https://t.co/W4R8Cu1xr5
Base editing without DNA off-target effects:
This study reports on an improved dual-guide #glycosylase #BaseEditor
➡https://t.co/9EEzES5USV
#CRISPR #Cas9
5/VERVE-201 is composed of messenger RNA for an adenine #baseeditor & a guide RNA designed to target the ANGPTL3 gene packaged in @VerveTx’s proprietary lipid nanoparticle LNP delivery technology - GalNAc-LNP.
With an improved dual #cytosine and #adenine #BaseEditor, Zhang et al. target OsEPSPS for artificial #evolution, generating a novel allele conferring glyphosate tolerance in #rice.
https://t.co/JzpOla4JHY @wileyplantsci #JIPB #PlantSci #CropSci
10/@VerveTx has presented preclinical data from NHP models of HoFH, including an evaluation in wild-type NHPs - comparing the ability of GalNAc-LNP to deliver its ANGPTL3 #BaseEditor versus standard LNPs. $VERV GaINAc-LNPs reduced blood ANGPTL3 by 94%-97% (!) in NHP model! 🧵👇
NOW! Gene Targeting & Gene
Correction: New Technologies 515 AB #ASGCT2023 #ASGCT23 Shuttle Peptide Delivers #BaseEditor RNPs to Rhesus Monkey Airway Epithelial
Cells In Vivo #cysticfibrosis @somaticediting #CFawarenessmonth
Our weekly newsletter has just dropped! Enjoy your weekly one-stop shop for research and clinical updates from the therapeutic gene-editing field!
Check it out! ---> https://t.co/t9ax9dG0ix
#crisprmedicinenews #crisprmedicine #geneediting #talen #meganuclease #baseeditor
2/VERVE-201 is composed of messenger RNA for an adenine #baseeditor & a guide RNA designed to target the ANGPTL3 gene packaged in @VerveTx’s proprietary lipid nanoparticle #LNP #delivery #technology - GalNAc-LNP.
Cell and gene therapies is undoubtably making waves this year especially with #CRISPR and #baseeditor companies presenting positive interim clinical research in their phase1/2 trial.
In our newest clinical update, we look at four ongoing gene-editing trials for solid cancers.
Read it here: https://t.co/rlZrhilsb0
#crisprmedicinenews #crisprmedicine #crispr #geneediting #clinicaltrial #baseeditor #solidtumour
To mark the start of 2023, we look back on some of the key advances in gene-editing clinical trials from 2022.
Check it out! https://t.co/G5Q54oZkSL
#crisprmedicinenews #crisprmedicine #baseeditor #crispr #geneediting #talens #clinicalnews
Free seminar "Charting the activity landscape of DNMT3A through base editor scanning" with @nicklue8 @varianteffects #BaseEditor #DNMT3A
https://t.co/swg0iRswOE Staring momentarily!
🚨New Publication🚨 #SCGE PI @davidrliu and his lab @liugroup demonstrate how a cytosine #baseeditor can be developed into an adenine base editor, providing low off-target activity and high on-target activity https://t.co/nWNz7JOQEK
5/The $BEAM-101 #BEACON #trail is the first #ClinicalTrial of a #BaseEditor in the U.S. & is expected to include an initial “sentinel” cohort of three patients - treated one at a time in order to confirm successful engraftment, followed by dosing in up to a total of 45 #patients.
A newly developed next-generation adenine base editor may improve the safety of human gene therapy.
#genomeediting #baseeditor #genetherapy #crispr #geneediting
6/@VerveTx has presented an updated data from NHP models of HoFH, including an evaluation in wild-type NHPs - comparing the ability of GalNAc-LNP to deliver its ANGPTL3 #BaseEditor versus standard LNPs. $VERV GaINAc-LNPs reduced blood ANGPTL3 by 94%-97% (!) in NHP model! 🧵👇
The first #HeterozygousFamilialHypercholesterolemia patient has been dosed with @VerveTx's single-dose PCSK9 adenine #baseeditor in a PhIb #clinical trial. #GTInTheWorld
📸: ©iStock
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