Taking the stage now is Mark Dawson of the Peter MacCallum Cancer Centre. His talk is about understanding human pathology with base editing at single-cell resolution.
#VariantEffect26
#BaseEditing
#AtlasOfVariantEffectsAlliance
#AHAS multi-gene #baseediting
nCas9-PmCDA1 → GmAHAS2/3/4 editing
6 lines: P182S/Y, triple-stacked
2S3S4F (triple) → strongest PE resistance (27.99cm, 2.24g vs. WT 9.13cm, 0.17g)
2Y → BS-specific (binding 7.734→6.766 kcal/mol)
2Y3SC → dual PE+BS
Retains IM resistance #Herbicide
#BaseEditing is still a relatively new #biotechnology. Researchers are working to improve its efficiency, while lowering the incidence of unwanted bystander edits. Alexis Komor’s lab has developed a new tool to do this. Their work appears in Nature Biotechnology https://bit.ly/47IKYHB
#TGFBI #baseediting for #cornealdystrophy
#ABE8e-NG: R124C 91%, R555W 62%
Allele-specific, <0.2% indels, <0.7% bystander, <1.2% off-target
Function restored: TGFBI lysosomal mislocalization reversed
AAV1 = best corneal transduction
Dominant correction ✓
doi.org/10.1167/iovs...
A phase 1 study shows feasibility of PCSK9 #BaseEditing for familial hypercholesterolemia, but clinical translation will require optimal editing , safety, patient selection & trial design.
News & Views from Robert Rosenson, Sascha Goonewardena
https://www.nature.com/articles/s41591-026-04220-0
🐟 Codon-optimized base editors enable efficient A→G & C→T editing in Atlantic salmon
ssABE8e & ssCBE4max-SpRY outperform unoptimized versions in ASK cells & embryos.
tyr KO → albino phenotype. Multiple loci validated.
www.cell.com/trends/biote...
#BaseEditing #Aquaculture #CRISPR
New therapeutic Adenine Base Editor (ABE) with minimized off-target effects! 🧬
A study in Protein&Cell by Erwei Zuo’s team introduces ABE8eY149V.
Read more: doi.org/10.1093/proc...
#GeneEditing #BaseEditing #Biotech #sciencenews
Anyone attending #Deaminet2026 on January 21-23? Looking forward to catching up on the latest developments in #baseediting and #primeediting!
labs.uthscsa.edu/rsh/deaminet...
@addgene.bsky.social will be sponsoring poster prizes!
#CRISPR #geneediting #GeneAndCellTherapy #CellAndGeneTherapy
Latest research from my group. We activated BDNF protein expression by disrupting a repressive uORF using a CRISPR base editing strategy: www.sciencedirect.com/science/arti...
#OxfordPaediatrics #IDRMOxford #BDNF #uORF #CRISPR #BaseEditing
🌟New hope for vision restoration: Congratulations to Bence György on his 5-year @erc.europa.eu Consolidator Grant. His proposal was selected among 3121 applications from across Europe - a strong vote of confidence in our work at IOB!
#ERCCoG #BaseEditing #VisionResearch #Blindness #AcademicBlueSky
🌟New hope for vision restoration: Warm congratulations to Bence György on his 5-year @erc.europa.eu Consolidator Grant to advance base editing for genetic macular degeneration and explore therapies for corneal blindness.
#ERCCoG #BaseEditing #VisionResearch #Blindness #AcademicBlueSky
It's always exciting to see pioneering research making the leap into pioneering therapies that have had real long-term success:
www.bbc.co.uk/news/article...
#Research #BaseEditing
Our #BaseEditing paper is DG-GT’s “Paper of the Quarter”! 🎉
We used precise gene editing to correct cancer mutations in TP53 – and watched as corrected cells nearly vanished. A step toward mRNA therapies for cancer! 💡
Full paper: rdcu.be/exqtH #CancerResearch #GeneEditing #Science #DG_GT
This review highlights recent advances in #Mitochondrial #BaseEditing & its relevance to #NeurodegenerativeDiseases, offering insights into how this technology could inform future strategies for prevention & targeted intervention. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
This paper was truly a team effort, thanks to all who came together and made it happen! #CRISPR #GeneEditing #GenomeEditing #BaseEditing #AdenineBaseEditing
Baby KJ; doctors will ask FDA this winter to OK a clinical trial of pioneering gene editing approach.
#genetherapy
#baseediting
#crispr
#geneediting
www.statnews.com/2025/10/16/b... via @statnews.com
Chao and colleagues show an image of glioblastoma (red) treated with CF17 oncolytic virus (green). Image credit: Jianfei Chao.
#MolecularTherapy Oncology's Sept 2025 issue covers new research on #Nanobodies, #OncolyticViruses, #BaseEditing, and #TherapeuticTargets in #Cancer. Check it out today: http://dlvr.it/TN8jsh
#ASGCT
Diagram illustrating how precise editing of the ZmACC1/2 genes generated herbicide-tolerant maize germplasm, offering a solution for weed management in maize-soybean intercropping systems.
🌽#Biotech breakthrough!🧬
Robust #genetic mods without yield loss? Fu et al. show it's possible in new work that enhances #herbicide tolerance in #maize using cytosine and adenine #BaseEditing!
Read the entire article for #free right here!⬇️
doi.org/10.1111/jipb...
#PlantScience #OpenAccess
Read the full study here:
rdcu.be/exqtH
Lastly, this wouldn't have been possible without a fantastic team of co-authors ☺️ Truly grateful to everyone who contributed and supported the work along the way.
#BaseEditing #PrecisionOncology #TP53 #CRISPR
#baseEditing #personalizedMedicine
#precisionGeneEditing
We are there!! 💛🧬
As of June, at least 20 clinical trials with base or prime editors are underway. The newer base and prime editing medicines modify or insert specific nucleotides into the DNA without creating double-stranded breaks.
Proud of Kaisa Pakari @kpakari.bsky.social to receive the 2025 Schmeil young researcher award for her #PhD thesis. She established 🐟 models to address disorders of #glycosylation #CDG by advancing #baseediting range for precise genome editing @wittbrodtlab.bsky.social @cos.uni-heidelberg.de
Correction of m.4291T > C in primary patient-derived fibroblasts. Top left: The primary patient-derived fibroblast line is homoplasmic for the m.4291T > C mutation. Top right: Design of two sets of Left (red) and Right (green) target sequences flanking the spacer region (blue) for TALE-guided editing in MT-TI to correct m.4291C > T (dark red). Mismatched T (red) in TALE target sequence L2. Middle: Overview of the transfection strategy of DdCBE for primary skin fibroblasts. Bottom left: Editing efficiencies of all four combinations Left- and Right-TALE-DdCBE plasmids in primary patient-derived fibroblasts as measured by amplicon sequencing (Illumina NGS). Bottom middle: Ratio of on-target editing vs. bystander editing at any other base within the spacing region in fibroblast cells. Bottom right: Clonal lines of edited primary patient-derived fibroblasts show high variability in mitochondrial heteroplasmy indicating different editing efficiencies per cell (Illumina NGS).
What is the therapeutic potential of correcting mutations by base editing of the #MitochondrialGenome? This study shows that #mitochondrial #BaseEditing can indeed functionally create & correct mitochondrial pathogenic #mutations in patient-derived cells @plosbiology.org 🧪 plos.io/3ZMa2K2
Correction of m.4291T > C in primary patient-derived fibroblasts. Top left: The primary patient-derived fibroblast line is homoplasmic for the m.4291T > C mutation. Top right: Design of two sets of Left (red) and Right (green) target sequences flanking the spacer region (blue) for TALE-guided editing in MT-TI to correct m.4291C > T (dark red). Mismatched T (red) in TALE target sequence L2. Middle: Overview of the transfection strategy of DdCBE for primary skin fibroblasts. Bottom left: Editing efficiencies of all four combinations Left- and Right-TALE-DdCBE plasmids in primary patient-derived fibroblasts as measured by amplicon sequencing (Illumina NGS). Bottom middle: Ratio of on-target editing vs. bystander editing at any other base within the spacing region in fibroblast cells. Bottom right: Clonal lines of edited primary patient-derived fibroblasts show high variability in mitochondrial heteroplasmy indicating different editing efficiencies per cell (Illumina NGS).
What is the therapeutic potential of correcting mutations by base editing of the #MitochondrialGenome? This study shows that #mitochondrial #BaseEditing can indeed functionally create & correct mitochondrial pathogenic #mutations in patient-derived cells @plosbiology.org 🧪 plos.io/3ZMa2K2
Correction of m.4291T > C in primary patient-derived fibroblasts. Top left: The primary patient-derived fibroblast line is homoplasmic for the m.4291T > C mutation. Top right: Design of two sets of Left (red) and Right (green) target sequences flanking the spacer region (blue) for TALE-guided editing in MT-TI to correct m.4291C > T (dark red). Mismatched T (red) in TALE target sequence L2. Middle: Overview of the transfection strategy of DdCBE for primary skin fibroblasts. Bottom left: Editing efficiencies of all four combinations Left- and Right-TALE-DdCBE plasmids in primary patient-derived fibroblasts as measured by amplicon sequencing (Illumina NGS). Bottom middle: Ratio of on-target editing vs. bystander editing at any other base within the spacing region in fibroblast cells. Bottom right: Clonal lines of edited primary patient-derived fibroblasts show high variability in mitochondrial heteroplasmy indicating different editing efficiencies per cell (Illumina NGS).
What is the therapeutic potential of correcting mutations by base editing of the #MitochondrialGenome? This study shows that #mitochondrial #BaseEditing can indeed functionally create & correct mitochondrial pathogenic #mutations in patient-derived cells @plosbiology.org 🧪 plos.io/3ZMa2K2
🧬 EFMC Session at #ChemBioParis2025
"Genome Base Editing & Precision Chemistry for Therapy"
Explore next-gen therapeutics with Claudia Höbartner & Chase Beisel
🎙 Organized by @euromedchem.bsky.social
📍 Paris | 🗓 Oct 6–9, 2025
🔗 chembioparis2025.com
#GeneEditing #BaseEditing #ChemBio #MedChem
We’re sure you saw the big news last week — personalized CRISPR just saved a baby’s life.
But how did the scientists do it?
In our latest CRISPR180", Antonio Carusillo and Manel Llado break down this amazing research for you in just 3 minutes.
#CRISPR #BaseEditing #RareDisease #GeneEditing
Masako Kaufmann and her certificate with the awarded poster on universal CAST-Seq
Highlighst of the 2025 ThemeDay:
Congratulations to Masako Kaufmann @uni-freiburg.de for the best poster at the #DGGTeV #Themeday2025 for her work on #BaseEditing #offtarget detection via universal CAST-Seq
Exciting opportunity for @astarsg.bsky.social student @cggoh.bsky.social to exchange new ideas on #CRISPR and #BaseEditing with @spjacksongroup.bsky.social alumnus Dan Durocher @durocher1.bsky.social, with postdoc Dr Simon Lam at #CRISPR25 @eventswcs.bsky.social
I am ecstatic to share this work, which involved functional interrogation of MUTYH variants using base editing. I enjoyed working with Dr. Carlos Vasquez on this project and look forward to seeing this expanded for high throughput!
#baseediting #womeninstem 🧪
academic.oup.com/nar/article/...
Following on the heels of #VerveTherapeutics with VERVE-102, #BeamTherapeutics also got IND clearance from FDA for its #BaseEditing therapeutic: finance.yahoo.com/news/beam-th...
