🚀 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
October marks #RettSyndromeAwarenessMonth, a time to shine a light on Rett syndrome.
At ProQR, we are proud to partner with the Rett Syndrome Research Trust developing #Axiomer #RNAEditing oligonucleotides to target genetic variants that cause Rett syndrome.
At ProQR, we are proud to partner with the Rett Syndrome Research Trust developing #Axiomer #RNAEditing oligonucleotides to target genetic variants that cause Rett syndrome.
October 22, 2025 at 1:05 PM
October marks #RettSyndromeAwarenessMonth, a time to shine a light on Rett syndrome.
At ProQR, we are proud to partner with the Rett Syndrome Research Trust developing #Axiomer #RNAEditing oligonucleotides to target genetic variants that cause Rett syndrome.
At ProQR, we are proud to partner with the Rett Syndrome Research Trust developing #Axiomer #RNAEditing oligonucleotides to target genetic variants that cause Rett syndrome.
Adenosine-to-inosine editing of miR-200b-3p is associated with the progression of high-grade serous ovarian cancer
👉 buff.ly/FMrZvtO
#HGSOC #miR200 #RNAediting
👉 buff.ly/FMrZvtO
#HGSOC #miR200 #RNAediting
September 1, 2025 at 8:33 AM
Adenosine-to-inosine editing of miR-200b-3p is associated with the progression of high-grade serous ovarian cancer
👉 buff.ly/FMrZvtO
#HGSOC #miR200 #RNAediting
👉 buff.ly/FMrZvtO
#HGSOC #miR200 #RNAediting
Lemelinda pairs her passion for RNA biology with research, protocol development & biosafety support, while also fostering a strong community at ProQR through our Coffee & Collaboration Café.
Meet more of the ProQR team at: bit.ly/45ZFtmq
#Axiomer #RNAEditing #TeamProQR
Meet more of the ProQR team at: bit.ly/45ZFtmq
#Axiomer #RNAEditing #TeamProQR
August 26, 2025 at 9:36 AM
Lemelinda pairs her passion for RNA biology with research, protocol development & biosafety support, while also fostering a strong community at ProQR through our Coffee & Collaboration Café.
Meet more of the ProQR team at: bit.ly/45ZFtmq
#Axiomer #RNAEditing #TeamProQR
Meet more of the ProQR team at: bit.ly/45ZFtmq
#Axiomer #RNAEditing #TeamProQR
David, Senior Scientist at ProQR, plays a key role in advancing cell biology research while also contributing to team leadership and workplace culture.
Meet the rest of our team at: bit.ly/4nbVZWR
#Axiomer #RNAEditing #TeamProQR
Meet the rest of our team at: bit.ly/4nbVZWR
#Axiomer #RNAEditing #TeamProQR
October 15, 2025 at 9:47 AM
David, Senior Scientist at ProQR, plays a key role in advancing cell biology research while also contributing to team leadership and workplace culture.
Meet the rest of our team at: bit.ly/4nbVZWR
#Axiomer #RNAEditing #TeamProQR
Meet the rest of our team at: bit.ly/4nbVZWR
#Axiomer #RNAEditing #TeamProQR
🔗 In-depth analysis of the RNA editing landscape in intracranial aneurysms and its potential role in alternative splicing. Computational and Structural Biotechnology Journal, DOI: doi.org/10.1016/j.cs...
📚 CSBJ: www.csbj.org
#RNAediting #Neuroscience #Genomics #PrecisionMedicine #BrainHealth
📚 CSBJ: www.csbj.org
#RNAediting #Neuroscience #Genomics #PrecisionMedicine #BrainHealth
September 30, 2025 at 4:41 AM
🔗 In-depth analysis of the RNA editing landscape in intracranial aneurysms and its potential role in alternative splicing. Computational and Structural Biotechnology Journal, DOI: doi.org/10.1016/j.cs...
📚 CSBJ: www.csbj.org
#RNAediting #Neuroscience #Genomics #PrecisionMedicine #BrainHealth
📚 CSBJ: www.csbj.org
#RNAediting #Neuroscience #Genomics #PrecisionMedicine #BrainHealth
We’re excited to share that our CTA for the Phase 1 study of AX-0810 in healthy volunteers has been authorized!
Join our virtual Investor and Analyst Event Nov 3, 10:00 ET.
For more details, check out the PR: bit.ly/3LjRCLX
#Axiomer #RNAEditing
Join our virtual Investor and Analyst Event Nov 3, 10:00 ET.
For more details, check out the PR: bit.ly/3LjRCLX
#Axiomer #RNAEditing
October 20, 2025 at 12:48 PM
We’re excited to share that our CTA for the Phase 1 study of AX-0810 in healthy volunteers has been authorized!
Join our virtual Investor and Analyst Event Nov 3, 10:00 ET.
For more details, check out the PR: bit.ly/3LjRCLX
#Axiomer #RNAEditing
Join our virtual Investor and Analyst Event Nov 3, 10:00 ET.
For more details, check out the PR: bit.ly/3LjRCLX
#Axiomer #RNAEditing
#MC4R certainly shows potential. Will have to see detailed data. For a real medicine though, less frequent, possibly more specificity needed. Or could #RNAediting by mimicking GWAS variants be more potent+safe?
March 31, 2025 at 2:14 PM
#MC4R certainly shows potential. Will have to see detailed data. For a real medicine though, less frequent, possibly more specificity needed. Or could #RNAediting by mimicking GWAS variants be more potent+safe?
Sepul Bio, Doses First Participant in Phase 2b Clinical Trial of ultevursen for USH2A-associated retinitis pigmentosa. More information on sepulbio.com #IRD #RP #RNAediting
Sepul Bio : Transformative RNA therapies for inherited retinal diseases - Théa
Sepul Bio is a pioneering company in the field of transformative RNA therapies, specializing in rare genetic eye diseases.
Sepulbio.com
December 17, 2024 at 8:39 AM
Sepul Bio, Doses First Participant in Phase 2b Clinical Trial of ultevursen for USH2A-associated retinitis pigmentosa. More information on sepulbio.com #IRD #RP #RNAediting
Today we announced our First Quarter Operating and Financial Results.
Read the press release here: bit.ly/4dhMSRe
$PRQR #RNAEditing #Axiomer
Read the press release here: bit.ly/4dhMSRe
$PRQR #RNAEditing #Axiomer
May 8, 2025 at 11:11 AM
Today we announced our First Quarter Operating and Financial Results.
Read the press release here: bit.ly/4dhMSRe
$PRQR #RNAEditing #Axiomer
Read the press release here: bit.ly/4dhMSRe
$PRQR #RNAEditing #Axiomer
As R&D Team Manager at ProQR, Lorenza drives scientific collaboration and project planning while also championing inclusivity as part of our DEI team.
Meet the ProQR team at: bit.ly/4m4oZzs
#Axiomer #RNAEditing #TeamProQR
Meet the ProQR team at: bit.ly/4m4oZzs
#Axiomer #RNAEditing #TeamProQR
July 22, 2025 at 8:45 AM
As R&D Team Manager at ProQR, Lorenza drives scientific collaboration and project planning while also championing inclusivity as part of our DEI team.
Meet the ProQR team at: bit.ly/4m4oZzs
#Axiomer #RNAEditing #TeamProQR
Meet the ProQR team at: bit.ly/4m4oZzs
#Axiomer #RNAEditing #TeamProQR
#ATTR-CM for the #RNAediting and #genomeediting aficionados, worth noting that the most common form of vATTR-CM in US+UK is one that can be fixed by A-->I/G editing.
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
May 17, 2025 at 1:50 PM
#ATTR-CM for the #RNAediting and #genomeediting aficionados, worth noting that the most common form of vATTR-CM in US+UK is one that can be fixed by A-->I/G editing.
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
ProQR is officially a Great Place to Work!
We’re proud to foster a culture where innovation, collaboration, and growth thrive—driven by our amazing team and our mission to develop transformative RNA therapies.
bit.ly/42LdvZR
#GPTW #Biotech #RNAEditing #Axiomer
We’re proud to foster a culture where innovation, collaboration, and growth thrive—driven by our amazing team and our mission to develop transformative RNA therapies.
bit.ly/42LdvZR
#GPTW #Biotech #RNAEditing #Axiomer
April 25, 2025 at 8:00 AM
ProQR is officially a Great Place to Work!
We’re proud to foster a culture where innovation, collaboration, and growth thrive—driven by our amazing team and our mission to develop transformative RNA therapies.
bit.ly/42LdvZR
#GPTW #Biotech #RNAEditing #Axiomer
We’re proud to foster a culture where innovation, collaboration, and growth thrive—driven by our amazing team and our mission to develop transformative RNA therapies.
bit.ly/42LdvZR
#GPTW #Biotech #RNAEditing #Axiomer
🔍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...
Patho-DBiT
Spatial #RNAWorld on FFPE section👹
Now tissues can be mapped by RNA variants or non-coding RNAs
Whole transcriptome seq covers
mRNA
LincRNA
microRNA
sn/snoRNA
#AlternativeSplicing
#RNAEditing
RNA #SingleNucleotideVariation
In situ polyA
bioRxiv 2024
www.biorxiv.org/content/10.1...
Spatial #RNAWorld on FFPE section👹
Now tissues can be mapped by RNA variants or non-coding RNAs
Whole transcriptome seq covers
mRNA
LincRNA
microRNA
sn/snoRNA
#AlternativeSplicing
#RNAEditing
RNA #SingleNucleotideVariation
In situ polyA
bioRxiv 2024
www.biorxiv.org/content/10.1...
February 9, 2024 at 1:06 PM
Patho-DBiT
Spatial #RNAWorld on FFPE section👹
Now tissues can be mapped by RNA variants or non-coding RNAs
Whole transcriptome seq covers
mRNA
LincRNA
microRNA
sn/snoRNA
#AlternativeSplicing
#RNAEditing
RNA #SingleNucleotideVariation
In situ polyA
bioRxiv 2024
www.biorxiv.org/content/10.1...
Spatial #RNAWorld on FFPE section👹
Now tissues can be mapped by RNA variants or non-coding RNAs
Whole transcriptome seq covers
mRNA
LincRNA
microRNA
sn/snoRNA
#AlternativeSplicing
#RNAEditing
RNA #SingleNucleotideVariation
In situ polyA
bioRxiv 2024
www.biorxiv.org/content/10.1...
#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/...
You know that #CRISPR has fallen on hard times...
...when ADAR guy feels like the star presenter at a CRISPR conference telling all about the advantages of oligo-based A-->I #RNAediting
....and Sangamo's #ZincFingers are accepted as equal.
...when ADAR guy feels like the star presenter at a CRISPR conference telling all about the advantages of oligo-based A-->I #RNAediting
....and Sangamo's #ZincFingers are accepted as equal.
April 10, 2025 at 4:56 PM
You know that #CRISPR has fallen on hard times...
...when ADAR guy feels like the star presenter at a CRISPR conference telling all about the advantages of oligo-based A-->I #RNAediting
....and Sangamo's #ZincFingers are accepted as equal.
...when ADAR guy feels like the star presenter at a CRISPR conference telling all about the advantages of oligo-based A-->I #RNAediting
....and Sangamo's #ZincFingers are accepted as equal.
My continued biological education
Epigenetic regulation of #Aging: implications for interventions of aging & diseases
Beyond DNA & Histone epigenetic clock
#RNAModification m6A m5C (Writer/Reader/Eraser) #RNAEditing in #Senescence
Noncoding RNA
www.nature.com/articles/s41...
Epigenetic regulation of #Aging: implications for interventions of aging & diseases
Beyond DNA & Histone epigenetic clock
#RNAModification m6A m5C (Writer/Reader/Eraser) #RNAEditing in #Senescence
Noncoding RNA
www.nature.com/articles/s41...
September 22, 2024 at 12:15 PM
My continued biological education
Epigenetic regulation of #Aging: implications for interventions of aging & diseases
Beyond DNA & Histone epigenetic clock
#RNAModification m6A m5C (Writer/Reader/Eraser) #RNAEditing in #Senescence
Noncoding RNA
www.nature.com/articles/s41...
Epigenetic regulation of #Aging: implications for interventions of aging & diseases
Beyond DNA & Histone epigenetic clock
#RNAModification m6A m5C (Writer/Reader/Eraser) #RNAEditing in #Senescence
Noncoding RNA
www.nature.com/articles/s41...
#RNAEditing $WVE is dosing 200mg every 2 weeks in ongoing #AATD trial. Single dose now at 400mg.
$PRQR $KRRO $BEAM
$PRQR $KRRO $BEAM
March 4, 2025 at 8:56 PM
#RNAEditing $WVE is dosing 200mg every 2 weeks in ongoing #AATD trial. Single dose now at 400mg.
$PRQR $KRRO $BEAM
$PRQR $KRRO $BEAM
#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!
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
Genetic editing holds promise to treat incurable diseases, but #CRISPR sometimes does more harm than good. @UCSanDiego.bsky.social and @Yale.edu researchers report a new, potentially safer method: small nuclear #RNAediting.
Small Nuclear RNA Base Editing a Safer Alternative to CRISPR, UC San Diego Researchers Find
Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale…
buff.ly
September 18, 2025 at 3:04 PM
Genetic editing holds promise to treat incurable diseases, but #CRISPR sometimes does more harm than good. @UCSanDiego.bsky.social and @Yale.edu researchers report a new, potentially safer method: small nuclear #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
I mentioned Prime and Beam because they can 'fix' genes with base and prime editing. Approach also applies to ADAR #RNAediting when targeted at individual mutations, e.g. in #RettSyndrome. #ProQR #WaveLifeSciences
May 15, 2025 at 6:56 PM
I mentioned Prime and Beam because they can 'fix' genes with base and prime editing. Approach also applies to ADAR #RNAediting when targeted at individual mutations, e.g. in #RettSyndrome. #ProQR #WaveLifeSciences
Great to connect with the local community at the Dutch Medicines Summer Afternoon!
Our VP Axiomer Science, Lenka van Sint Fiet, demonstrated how #Axiomer is driving progress toward potentially transformative therapies.
#RNAEditing #DutchBioScienceWeek
Our VP Axiomer Science, Lenka van Sint Fiet, demonstrated how #Axiomer is driving progress toward potentially transformative therapies.
#RNAEditing #DutchBioScienceWeek
July 25, 2025 at 11:42 AM
Great to connect with the local community at the Dutch Medicines Summer Afternoon!
Our VP Axiomer Science, Lenka van Sint Fiet, demonstrated how #Axiomer is driving progress toward potentially transformative therapies.
#RNAEditing #DutchBioScienceWeek
Our VP Axiomer Science, Lenka van Sint Fiet, demonstrated how #Axiomer is driving progress toward potentially transformative therapies.
#RNAEditing #DutchBioScienceWeek