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Base editing milestone for familial hypercholesterolemia - Nature Medicine A phase 1 study demonstrates proof-of-concept of liver-targeted PCSK9 base editing for familial hypercholesterolemia, but turning this into a transformative therapy will require stringent optimization of editing efficiency, safety, patient selection and trial design.

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

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🐟 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

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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

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Deaminet 2026 - Harris Lab

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

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Targeted BDNF upregulation via upstream open reading frame disruption To understand the relative contributions of 5ʹ UTR elements to translation output, we performed a comprehensive analysis of upstream open reading fram…

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

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🌟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

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🌟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

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Blood cancer therapy reverses incurable leukaemia in some patients Seven out of 11 patients with incurable cancer who had the treatment appear to be cancer-free.

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

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A base editing platform for the correction of cancer driver mutations unmasks conserved p53 transcription programs

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

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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...

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This paper was truly a team effort, thanks to all who came together and made it happen! #CRISPR #GeneEditing #GenomeEditing #BaseEditing #AdenineBaseEditing

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Baby KJ's doctors will ask FDA this winter to OK a clinical trial of pioneering gene editing approach Baby KJ's medical team intend to ask the FDA to start a clinical trial that would be open to patients with seven different urea cycle disorders.

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

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Chao and colleagues show an image of glioblastoma (red) treated with CF17 oncolytic virus (green). Image credit: Jianfei Chao.

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

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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.

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

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A base editing platform for the correction of cancer driver mutations unmasks conserved p53 transcription programs

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

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Precision gene editing medicine makes history, and it’s just getting started - Nature Biotechnology Despite their astounding success, custom-made base editors and prime editors will need time to broaden their clinical impact.

#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.

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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

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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).

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

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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).

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

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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).

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

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🧬 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

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Personalized CRISPR just saved a baby's life
Personalized CRISPR just saved a baby's life YouTube video by WeDoCRISPR

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

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Masako Kaufmann and her certificate with the awarded poster on universal CAST-Seq

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

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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

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Precision genome editing and in-cell measurements of oxidative DNA damage repair enable functional and mechanistic characterization of cancer-associated MUTYH variants Abstract. Functional characterization of genetic variants has the potential to advance the field of precision medicine by enhancing the efficacy of current

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/...

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Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administ... U.S. IND Represents Regulatory Clearance For BEAM-302 Spanning Six Countries and Second Open IND for a Beam In Vivo Base Editing Program in the U.S. CAMBRIDGE, Mass., March 27, 2025 (GLOBE NEWSWIRE) -...

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...

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CRISPR im klinischen Kontext: Wie CRISPR die Medizin verändert Die Entdeckung und Weiterentwicklung der CRISPR-Technologie hat das Feld der Genomeditierung grundlegend verändert. Ursprünglich als adaptives Immunsystem von Bakterien identifiziert, wurde das CRISPR...

CRISPR ist in der Klinik angekommen – aber nur ein Produkt ist bislang zugelassen.
Was steckt hinter der Technologie und wer treibt sie voran?

🧬 Überblick zu #Casgevy, Base & Prime Editing, Unternehmen & Trends:
👉 www.blaubiologie.de/crispr-im-kl...

#CRISPR #Biotech #GeneEditing #Cas9 #BaseEditing

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Dimerization of the deaminase domain and locking interactions with Cas9 boost base editing efficiency in ABE8e Abstract. CRISPR-based DNA adenine base editors (ABEs) hold remarkable promises to address human genetic diseases caused by point mutations. ABEs were deve

Do you want to know why #ABE 8e is the most efficient adenine base editor? Read our paper with the amazing
Audrey Lapinaite!
@narjournal.bsky.social

The dimerization of TadA8e and its exclusive interactions with #CRISPR Cas9 boost #BaseEditing efficiency!
academic.oup.com/nar/article/...

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Genome editing with programmable base editors in human cells Genome editing has garnered significant attention over the last decade, resulting in a massive expansion of the genome engineering toolbox. Base edito…

🧪I am thrilled to share that our work was just published in Methods in Enzymology!

HUGE thank you to all who contributed to this, but especially to my phenomenal co-first author, @natalie-z.bsky.social, and our mentor, Dr. Alexis Komor.
#baseediting #genomeediting #SciSky #AcademicSky

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Engineering a bacterial toxin deaminase from the DYW-family into a novel cytosine base editor for plants and mammalian cells

I am very happy to share our latest manuscript in BMC Genome Biology 🥳: rdcu.be/d8weC We describe the engineering of the cytosine deaminase SsdA to build an efficient and small CRISPR/Cas9 base editor. #BaseEditing #Cas9 #CRISPR #GeneTherapy #GeneEditing Here is a short intro to a fun project 🧵🧪🧬

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