www.youtube.com/watch?v=ZPbf...
#sciencematters #sciencesavelives #pigorgan #geneediting #DNAediting #RNAediting #scientific
#sciencematters #sciencesavelives #pigorgan #geneediting #DNAediting #RNAediting #scientific
Judy Plus (119) The only person in the world living with a pig organ#geneediting #sciencesaveslives
YouTube video by Judy Plus--scientific discoveries.
www.youtube.com
March 23, 2025 at 9:39 AM
I’m excited that this review co-written with my colleague and friend Carl Walkley is now published! We focused on genetic studies, from mouse to human, that uncover the functional importance and mechanisms of RNA editing enzyme ADAR1 and reveal exciting therapeutic potential. #ADAR #RNAediting
New online! Leveraging genetics to understand ADAR1-mediated RNA editing in health and disease
Leveraging genetics to understand ADAR1-mediated RNA editing in health and disease
Nature Reviews Genetics, Published online: 14 April 2025; doi:10.1038/s41576-025-00830-5ADAR1-mediating RNA editing enables the cell to distinguish between endogenous and viral RNA. Li and Walkley review findings from human and mouse genetics that have revealed the mechanisms of ADAR1-mediated RNA editing, which are now providing insights for the development of potential therapies that target these mechanisms.
www.nature.com
April 15, 2025 at 1:45 AM
I’m excited that this review co-written with my colleague and friend Carl Walkley is now published! We focused on genetic studies, from mouse to human, that uncover the functional importance and mechanisms of RNA editing enzyme ADAR1 and reveal exciting therapeutic potential. #ADAR #RNAediting
#RNAediting patent app by #ProQR targeting gain-of-function in MC4R in the #CNS in #obesity.
patentscope.wipo.int/search/en/de...
patentscope.wipo.int/search/en/de...
March 25, 2025 at 9:31 PM
#RNAediting patent app by #ProQR targeting gain-of-function in MC4R in the #CNS in #obesity.
patentscope.wipo.int/search/en/de...
patentscope.wipo.int/search/en/de...
🌍 Calling all early-career RNA researchers! Join us in Lucca, Italy, March 15-16, 2025, for the RNA Editing Gordon Research Seminar (GRS)! 📅 #EarlyCareerScientists #RNAediting #RNAmodifications #RNAsky
December 2, 2024 at 4:25 PM
🌍 Calling all early-career RNA researchers! Join us in Lucca, Italy, March 15-16, 2025, for the RNA Editing Gordon Research Seminar (GRS)! 📅 #EarlyCareerScientists #RNAediting #RNAmodifications #RNAsky
#ProQR impressive preclinical AX-0810 cholestatic disease #ASGCT25 abstract. Clinical candidate shows >50% #RNAediting.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
May 13, 2025 at 9:49 AM
#ProQR impressive preclinical AX-0810 cholestatic disease #ASGCT25 abstract. Clinical candidate shows >50% #RNAediting.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
Very satisfying to see progress on #RNAediting efficiencies. Also >50% in #RettSyndrome program. Just 2 years ago, 1-3% were top hits without ADAR overexpression.
#ProQR
#ProQR
May 13, 2025 at 10:00 AM
Very satisfying to see progress on #RNAediting efficiencies. Also >50% in #RettSyndrome program. Just 2 years ago, 1-3% were top hits without ADAR overexpression.
#ProQR
#ProQR
#RNAEditing #PulmonaryArterialHypertension
ADAR1 (both p150 & p110 isoforms) ⬇️in 👤PAH (in 🫁vascular smooth muscle cell)
SMC ADAR1 KO🐭 excerbates PAH
SMC ⏫dsRNA➡️⏫PKR-eIF2B-IFNβ➡️
Macrophage M1 polarization➡️
SMC proliferation
@kekeyuan.bsky.social #CircRes 2025
www.ahajournals.org/doi/10.1161/...
ADAR1 (both p150 & p110 isoforms) ⬇️in 👤PAH (in 🫁vascular smooth muscle cell)
SMC ADAR1 KO🐭 excerbates PAH
SMC ⏫dsRNA➡️⏫PKR-eIF2B-IFNβ➡️
Macrophage M1 polarization➡️
SMC proliferation
@kekeyuan.bsky.social #CircRes 2025
www.ahajournals.org/doi/10.1161/...
November 23, 2025 at 12:37 PM
#RNAEditing #PulmonaryArterialHypertension
ADAR1 (both p150 & p110 isoforms) ⬇️in 👤PAH (in 🫁vascular smooth muscle cell)
SMC ADAR1 KO🐭 excerbates PAH
SMC ⏫dsRNA➡️⏫PKR-eIF2B-IFNβ➡️
Macrophage M1 polarization➡️
SMC proliferation
@kekeyuan.bsky.social #CircRes 2025
www.ahajournals.org/doi/10.1161/...
ADAR1 (both p150 & p110 isoforms) ⬇️in 👤PAH (in 🫁vascular smooth muscle cell)
SMC ADAR1 KO🐭 excerbates PAH
SMC ⏫dsRNA➡️⏫PKR-eIF2B-IFNβ➡️
Macrophage M1 polarization➡️
SMC proliferation
@kekeyuan.bsky.social #CircRes 2025
www.ahajournals.org/doi/10.1161/...
Axiomer Insights: Our team published findings in Scientific Reports on preclinical liver models for ADAR-mediated RNA editing. Monica from our research team discusses how model selection can inform translatability for our #Axiomer #RNAEditing platform.
Read the paper here: bit.ly/3UWJLZz
Read the paper here: bit.ly/3UWJLZz
September 25, 2026 at 12:03 PM
Axiomer Insights: Our team published findings in Scientific Reports on preclinical liver models for ADAR-mediated RNA editing. Monica from our research team discusses how model selection can inform translatability for our #Axiomer #RNAEditing platform.
Read the paper here: bit.ly/3UWJLZz
Read the paper here: bit.ly/3UWJLZz
#ProQR another nice gain-of-function #RNAediting patent app, this time inspired by a human genetic variant in the #reelin gene conferring 'extreme resistance' to #Alzheimer's.
April 3, 2025 at 6:17 AM
#ProQR another nice gain-of-function #RNAediting patent app, this time inspired by a human genetic variant in the #reelin gene conferring 'extreme resistance' to #Alzheimer's.
🚀 New publication from the team led by Kamel Hammani in Nucleic Acid Research ! They engineered synthetic RNA editors to precisely rewrite organellar RNAs in plants.
🔗 academic.oup.com/nar/article/...
#PlantBiotech #RNAediting #Chloroplast
🔗 academic.oup.com/nar/article/...
#PlantBiotech #RNAediting #Chloroplast
De novo RNA base editing in plant organelles with engineered synthetic P-type PPR editing factors
Abstract. In plant mitochondria and chloroplasts, cytidine-to-uridine RNA editing is necessary for the production of functional proteins. While natural PLS
academic.oup.com
April 10, 2025 at 7:58 PM
🚀 New publication from the team led by Kamel Hammani in Nucleic Acid Research ! They engineered synthetic RNA editors to precisely rewrite organellar RNAs in plants.
🔗 academic.oup.com/nar/article/...
#PlantBiotech #RNAediting #Chloroplast
🔗 academic.oup.com/nar/article/...
#PlantBiotech #RNAediting #Chloroplast
#Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
June 2, 2025 at 3:04 PM
#Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
🔍From #IL6 in #BipolarDisorder 🧬 to #RNAediting + #AI 🤖 diagnosing #Schizophrenia—science is bridging bench to bedside 💡 @mdpiopenaccess.bsky.social #Neuroimmunology #Biomarkers www.mdpi.com/1422-0067/26...
October 30, 2025 at 11:31 AM
🔍From #IL6 in #BipolarDisorder 🧬 to #RNAediting + #AI 🤖 diagnosing #Schizophrenia—science is bridging bench to bedside 💡 @mdpiopenaccess.bsky.social #Neuroimmunology #Biomarkers www.mdpi.com/1422-0067/26...
#CRISPR #RNAEditing #HypertrophicCardioMyopathy
AAV9-hpCas13d/gRNA
⏬30% RQ mutant mRNA
Partially rescue cardiac hypercontractility, LV hypertrophy & cardiomyocyte loss in early onset HCM of 2-mo Myh6 p.R872H/p.R404Q🐭
Resolve multiple🧍MYH7 variants
#CircAHA 2024
www.ahajournals.org/doi/10.1161/...
AAV9-hpCas13d/gRNA
⏬30% RQ mutant mRNA
Partially rescue cardiac hypercontractility, LV hypertrophy & cardiomyocyte loss in early onset HCM of 2-mo Myh6 p.R872H/p.R404Q🐭
Resolve multiple🧍MYH7 variants
#CircAHA 2024
www.ahajournals.org/doi/10.1161/...
April 1, 2025 at 12:01 PM
#CRISPR #RNAEditing #HypertrophicCardioMyopathy
AAV9-hpCas13d/gRNA
⏬30% RQ mutant mRNA
Partially rescue cardiac hypercontractility, LV hypertrophy & cardiomyocyte loss in early onset HCM of 2-mo Myh6 p.R872H/p.R404Q🐭
Resolve multiple🧍MYH7 variants
#CircAHA 2024
www.ahajournals.org/doi/10.1161/...
AAV9-hpCas13d/gRNA
⏬30% RQ mutant mRNA
Partially rescue cardiac hypercontractility, LV hypertrophy & cardiomyocyte loss in early onset HCM of 2-mo Myh6 p.R872H/p.R404Q🐭
Resolve multiple🧍MYH7 variants
#CircAHA 2024
www.ahajournals.org/doi/10.1161/...
Duan et al. propose the comparative RNA editomics analytical framework to prioritize potentially adaptive editing sites using multidimensional evidence, including condition specificity and cross-species evolutionary patterns.
doi.org/10.59717/j.t...
#RNAEditing #adaptation
doi.org/10.59717/j.t...
#RNAEditing #adaptation
September 22, 2026 at 5:04 AM
Duan et al. propose the comparative RNA editomics analytical framework to prioritize potentially adaptive editing sites using multidimensional evidence, including condition specificity and cross-species evolutionary patterns.
doi.org/10.59717/j.t...
#RNAEditing #adaptation
doi.org/10.59717/j.t...
#RNAEditing #adaptation
On Sept 30 at 11a ET join our virtual Investor and Analyst educational event “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing”.
For additional information about the event and to register, visit: www.proqr.com/events
#Axiomer #RNAEditing
For additional information about the event and to register, visit: www.proqr.com/events
#Axiomer #RNAEditing
September 25, 2026 at 8:17 PM
On Sept 30 at 11a ET join our virtual Investor and Analyst educational event “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing”.
For additional information about the event and to register, visit: www.proqr.com/events
#Axiomer #RNAEditing
For additional information about the event and to register, visit: www.proqr.com/events
#Axiomer #RNAEditing
🧪 What is RNA editing and why should we be excited about it?
My next article with @theconversation.bsky.social, this time with Rick Liu, my brilliant colleague at CERA, is out today! 🥳🗞️
#RNAEditing #GeneTherapy #WomenInSTEM 🧬 #SuperstarsOfSTEM
theconversation.com/rna-editing-...
My next article with @theconversation.bsky.social, this time with Rick Liu, my brilliant colleague at CERA, is out today! 🥳🗞️
#RNAEditing #GeneTherapy #WomenInSTEM 🧬 #SuperstarsOfSTEM
theconversation.com/rna-editing-...
RNA editing is the next frontier in gene therapy – here’s what you need to know
We can already tinker with human DNA to treat diseases. But being able to edit RNA allows for even more precision – and fewer risks.
theconversation.com
November 21, 2024 at 7:56 PM
🧪 What is RNA editing and why should we be excited about it?
My next article with @theconversation.bsky.social, this time with Rick Liu, my brilliant colleague at CERA, is out today! 🥳🗞️
#RNAEditing #GeneTherapy #WomenInSTEM 🧬 #SuperstarsOfSTEM
theconversation.com/rna-editing-...
My next article with @theconversation.bsky.social, this time with Rick Liu, my brilliant colleague at CERA, is out today! 🥳🗞️
#RNAEditing #GeneTherapy #WomenInSTEM 🧬 #SuperstarsOfSTEM
theconversation.com/rna-editing-...
Our recent edition of the Axiomer Summit brought insightful discussions, a dynamic poster session, and exciting platform updates.
Thanks to our SAB and Board for their valuable contributions!
#RNAEditing #Axiomer #Biotech
Thanks to our SAB and Board for their valuable contributions!
#RNAEditing #Axiomer #Biotech
December 22, 2025 at 9:35 AM
Our recent edition of the Axiomer Summit brought insightful discussions, a dynamic poster session, and exciting platform updates.
Thanks to our SAB and Board for their valuable contributions!
#RNAEditing #Axiomer #Biotech
Thanks to our SAB and Board for their valuable contributions!
#RNAEditing #Axiomer #Biotech
ProQR welcomes Chris Hart as Chief Data & AI Officer and Thomas Wolf as Strategic AI Advisor to the Board. Their appointments support our strategy to apply AI across discovery and development. AX-0811, the first AI-discovered ProQR program is now in clinical trials.
#Axiomer #RNAEditing
#Axiomer #RNAEditing
September 23, 2026 at 12:27 PM
ProQR welcomes Chris Hart as Chief Data & AI Officer and Thomas Wolf as Strategic AI Advisor to the Board. Their appointments support our strategy to apply AI across discovery and development. AX-0811, the first AI-discovered ProQR program is now in clinical trials.
#Axiomer #RNAEditing
#Axiomer #RNAEditing
Isn't this one of the promises of #RNAediting?
March 31, 2025 at 2:53 PM
Isn't this one of the promises of #RNAediting?
One good thing in the 'One Big Beautiful Bill' is that drugs with more than 1 #orphan indications will be excluded from IRA negotiations. This is relevant for #RNAi #RNAediting and #CRISPR targeting things like TTR, LDL-R, ApoB.
May 22, 2025 at 8:30 PM
One good thing in the 'One Big Beautiful Bill' is that drugs with more than 1 #orphan indications will be excluded from IRA negotiations. This is relevant for #RNAi #RNAediting and #CRISPR targeting things like TTR, LDL-R, ApoB.
Nice 80% #RNAediting efficiency in patient-derived cells at therapeutically relevant #oligonucleotide concentrations. If #ProQR can take advantage of #FDA platform pathway, #Rett opportunity very exciting.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
May 17, 2025 at 9:52 AM
Nice 80% #RNAediting efficiency in patient-derived cells at therapeutically relevant #oligonucleotide concentrations. If #ProQR can take advantage of #FDA platform pathway, #Rett opportunity very exciting.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
💡 Interested in light-controlled RNA engineering?
👉 Hanswillemenke et al. use light-controlled RNA-protein conjugates to regulate RNA base editing in living cells.
🔗 brnw.ch/21x5n6k
#RNAEditing #RNATargeting #Photocontrol
👉 Hanswillemenke et al. use light-controlled RNA-protein conjugates to regulate RNA base editing in living cells.
🔗 brnw.ch/21x5n6k
#RNAEditing #RNATargeting #Photocontrol
September 3, 2026 at 1:11 PM
💡 Interested in light-controlled RNA engineering?
👉 Hanswillemenke et al. use light-controlled RNA-protein conjugates to regulate RNA base editing in living cells.
🔗 brnw.ch/21x5n6k
#RNAEditing #RNATargeting #Photocontrol
👉 Hanswillemenke et al. use light-controlled RNA-protein conjugates to regulate RNA base editing in living cells.
🔗 brnw.ch/21x5n6k
#RNAEditing #RNATargeting #Photocontrol
Great to connect with the community at #TIDESEU in Basel last week. Gerard Platenburg, our Chief Scientific Officer, presented on translating #Axiomer RNA Editing science into targeted CNS applications.
Check out the presentation here: bit.ly/3K2y927
#RNAEditing
Check out the presentation here: bit.ly/3K2y927
#RNAEditing
November 21, 2025 at 2:27 PM
Great to connect with the community at #TIDESEU in Basel last week. Gerard Platenburg, our Chief Scientific Officer, presented on translating #Axiomer RNA Editing science into targeted CNS applications.
Check out the presentation here: bit.ly/3K2y927
#RNAEditing
Check out the presentation here: bit.ly/3K2y927
#RNAEditing
#RNA molecules are incredibly versatile, and one way they showcase this is through post-transcriptional modifications. These modifications expand the classic four-letter RNA alphabet (A, C, G, U) to include over 300 unique bases! 🌟
#RNAResearch #RNAEditing #LifeSciences
2/n
#RNAResearch #RNAEditing #LifeSciences
2/n
January 18, 2025 at 9:33 PM
#RNA molecules are incredibly versatile, and one way they showcase this is through post-transcriptional modifications. These modifications expand the classic four-letter RNA alphabet (A, C, G, U) to include over 300 unique bases! 🌟
#RNAResearch #RNAEditing #LifeSciences
2/n
#RNAResearch #RNAEditing #LifeSciences
2/n
🚨 New paper alert! 🚨
Our paper entitled: "Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3" is out now on Journal of Biological Chemistry!
www.jbc.org/article/S002...
#AlternativeSplicing #RNAediting
🧵 👇
Our paper entitled: "Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3" is out now on Journal of Biological Chemistry!
www.jbc.org/article/S002...
#AlternativeSplicing #RNAediting
🧵 👇
Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3
Alternative splicing and adenosine-to-inosine (A-to-I) RNA editing are post-transcriptional
mechanisms that promote proteomic diversity in the brain. The impact of RNA editing
on brain physiology is u...
www.jbc.org
October 14, 2025 at 2:34 PM
🚨 New paper alert! 🚨
Our paper entitled: "Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3" is out now on Journal of Biological Chemistry!
www.jbc.org/article/S002...
#AlternativeSplicing #RNAediting
🧵 👇
Our paper entitled: "Synergy between RNA editing and alternative splicing modulates the biological properties of the voltage-gated calcium channel CaV1.3" is out now on Journal of Biological Chemistry!
www.jbc.org/article/S002...
#AlternativeSplicing #RNAediting
🧵 👇