This study reveals FUS as a key driver of fibrotic signaling in #IdiopathicPulmonaryFibrosis, with #antisense therapy (ION363) reducing profibrotic gene programs & improving patient-derived lung models, highlighting FUS as a promising therapeutic target.

#STTT: doi.org/10.1038/s413...

`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`

www.science.org/doi/10.1126/scitranslmed...

#Rett #RettSyndrome #geneTherapy #antisense

Long-read #transcriptomics of #GallbladderCancer uncovered ERBB2 i14e, a splice isoform linked to poor prognosis and #trastuzumab resistance; #antisense targeting restored sensitivity, revealing a potential #biomarker and therapeutic strategy.

#STTT: doi.org/10.1038/s413...

How can markets be made for #SyntheticBiology products? Holland suggests #Innovation will accelerate once mechanisms are in place. bit.ly/4oXBUEJ #EngineeringBiology #Patents #TechnologyTransfer #PlasticDegradation #ChemicalManufacture #Therapeutics #DrugDelivery #Antisense #GeneExpression

Researchers establish a full-length #gallbladder #transcriptome atlas using #LongReadSequencing and identify ERBB2 i14e as a novel isoform driving #TumorGrowth and trastuzumab resistance. #ERBB2 #antisense #medsky

#OpenAccess in #STTT: doi.org/10.1038/s413...

Arnatar announces publication of key patents for ACT-UP1, a first-in-class #antisense platform to #upregulate protein expression. A breakthrough for #haploinsufficient & #ProteinDeficiency diseases.

🧬 Learn more: https://bit.ly/41JBRDE

Join us for an online seminar on ASO therapy, September 24th 12.30 pm
#RNAtherapeutics #Antisense
@gbmev.bsky.social @oligogirl.bsky.social

Our next speaker is Anne-Lot van Esbroeck from the @erasmusmc.bsky.social who is going to present on the development of #antisense therapies for brain diseases. In particular Rare Diseases, which the rarity of the disorders complicates significantly the chances of a treatment.

Thrilled to see our work on targeted #mRNA blockade using high-throughput #antisense screening, now published in Advanced Science! 🚀

A true team effort in close collaboration with the @jorg-vogel-lab.bsky.social with special credit to Popella & Danti!

ASOs for the people!
bsky.app/profile/gior...

#FWakeAntiSense #AntiSense
#FWakeYouTube #FranLab #FWakeFranLab /\
#FWakeYouTubeCommunity #CascadeComments /\
#FWakeAntiReality #AntiRealityOpera /\
#FWakeContextSense #FWakeTopicContext /\
#FWakeSpamFilters #FWakeSpamFilterBypass /\

www.youtube.com/watch?v=i91n...

Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease - Communications Medicine Karjosukarso et al. investigate antisense oligonucleotides targeting aberrant splicing caused by a recurrent ABCA4 variant in Stargardt disease. A candidate therapeutic molecule that shows a favorable...

Check out our latest work! Preclinical in vitro and safety and in vivo biodistribution of #antisense #oligonucleotides targeting a recurrent genetic variant in ABCA4 present in patients with #Stargardt disease. A big first step towards therapeutic intervention.
www.nature.com/articles/s43...

12 PhDs on Antisense oligonucleotide research to foster the full therapeutic potential of antisense technology across tissues EFFecT (European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues) is a Marie Skłodowska-Curie Doctoral Network (2025-2028), offering 12 PhD positions.

🚨 We're hiring! 🚨

Join us at Biobizkaia 🌍 as part of the MSCA-DN EffecT project! We’re offering a fully funded #PhD position UPV/EHU. 🎓

📌 Focus on:
🔬 #Antisense #oligonucleotides
🧬 #Splicing
🌟 #Rarediseases📄

Apply now: euraxess.ec.europa.eu/jobs/296420
Info here: www.effect-dn.eu/candidates

12 PhDs on Antisense oligonucleotide research to foster the full therapeutic potential of antisense technology across tissues EFFecT (European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues) is a Marie Skłodowska-Curie Doctoral Network (2025-2028), offering 12 PhD positions.

We're hiring!!
As part of the MSCA-DN EffecT we offer a PhD position in our lab at @CBM_CSIC_UAM @UAM_Madrid to work in #antisense oligonucleotides, #splicing and #rarediseases All the information here
euraxess.ec.europa.eu/jobs/296420