Improved cryo-EM reconstruction of sub-50 kDa complexes using 2D template matching Visualizing the structures of small proteins and complexes has been a longstanding challenge in single-particle cryo-EM. Some of these targets have been successfully resolved by binding to antibody fragments...

🧬 New BioRxiv preprint from the RTI: Improved cryo-EM reconstruction of sub-50 kDa complexes using 2D template matching buff.ly/2GRES82 #RNA #RNATherapeutics @umasschan.bsky.social

Hot off the press from the RTI: Bacteria producing the bioplastic polyhydroxybutyrate kill the nematode Caenorhabditis elegans

buff.ly/qiZ4LqH #RNA #RNATherapeutics @UMassChan

Bacteria producing the bioplastic polyhydroxybutyrate kill the nematode Caenorhabditis elegans - PubMed Bacteria, both individually and as symbionts of other organisms, significantly influence ecosystems by providing nutrients and metabolizing exogenous compounds. Some bacteria polymerize small organic...

Hot off the press from the RTI: Bacteria producing the bioplastic polyhydroxybutyrate kill the nematode Caenorhabditis elegans
pubmed.ncbi.nlm.nih.gov/41961891/?ut... #RNA #RNATherapeutics

Registration for the 8th annual RNA Therapeutics: From Concept to Clinic is filling fast and closing soon. Join us June 24–26, 2026 for three days of cutting-edge science and translational insights — secure your spot now: buff.ly/vi22rN6 #RNATherapeutics #RNATx2026 @UMassChan.bsky.social

Hot off the press from the RTI: Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells

buff.ly/UtkzwT5 #RNA #RNATherapeutics @UMassChan

Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells - PubMed Impaired angiogenesis is a common feature of several pathological conditions, including neuromuscular disorders. Such vascular defects not only contribute to disease progression but also may compromise...

Hot off the press from the RTI: Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells
pubmed.ncbi.nlm.nih.gov/41933450/?ut... #RNA #RNATherapeutics

Hot off the press from the RTI: Testis-specific lncRNA Teshl regulates acrosome biogenesis to maintain sperm structure and function

buff.ly/djTfcMt #RNA #RNATherapeutics @UMassChan

Testis-specific lncRNA Teshl regulates acrosome biogenesis to maintain sperm structure and function - PubMed Testis-specific lncRNA Teshl regulates acrosome biogenesis to maintain sperm structure and function

Hot off the press from the RTI: Testis-specific lncRNA Teshl regulates acrosome biogenesis to maintain sperm structure and function
pubmed.ncbi.nlm.nih.gov/41913266/?ut... #RNA #RNATherapeutics

The Approval of Redemplo for Familial Chylomicronemia Syndrome and the Many Flavors of GalNAc-Oligonucleotides - PubMed In the fourth quarter of 2025, a press release announced the approval of the eighth small interfering RNA (siRNA)-based therapeutic. Redemplo (plozasiran), developed by Arrowhead Pharmaceuticals, is…

Hot off the press from the Alterman & Khvorova labs: The Approval of Redemplo for Familial Chylomicronemia Syndrome and the Many Flavors of GalNAc-Oligonucleotides

buff.ly/chHxiUY #RNA #RNATherapeutics @UMassChan

Divalent siRNA for prion disease - PubMed Prion protein (PrP) lowering is effective in animal models of prion disease and is being tested clinically in prion disease patients, but there remains a need for more potent PrP-lowering drug candidates....

Hot off the press from the RTI: Divalent siRNA for prion disease
pubmed.ncbi.nlm.nih.gov/41867745/?ut... #RNA #RNATherapeutics

Post-Transcriptional Size-Dependent Expression of the Fission Yeast Cdc13 Cyclin The major fission yeast cyclin, Cdc13, has been shown to increase in concentration in correlation with cell size, and has been proposed to thereby regulate cell size at division. However, the mechanism...

🧬 New BioRxiv preprint from the RTI: Post-Transcriptional Size-Dependent Expression of the Fission Yeast Cdc13 Cyclin www.biorxiv.org/content/10.1... #RNA #RNATherapeutics @umasschan.bsky.social

Hot off the press from the RTI: RNAi-mediated p38δ silencing mitigates anthracycline cardiotoxicity in female mice

pubmed.ncbi.nlm.nih.gov/41874387/?ut... #RNA #RNATherapeutics @UMassChan

The Approval of Redemplo for Familial Chylomicronemia Syndrome and the Many Flavors of GalNAc-Oligonucleotides - PubMed In the fourth quarter of 2025, a press release announced the approval of the eighth small interfering RNA (siRNA)-based therapeutic. Redemplo (plozasiran), developed by Arrowhead Pharmaceuticals, is the...

Hot off the press from the RTI: The Approval of Redemplo for Familial Chylomicronemia Syndrome and the Many Flavors of GalNAc-Oligonucleotides
pubmed.ncbi.nlm.nih.gov/41877501/?ut... #RNA #RNATherapeutics

RNAi-mediated p38δ silencing mitigates anthracycline cardiotoxicity in female mice - PubMed The anthracycline antibiotic doxorubicin (DOX) is a potent chemotherapy drug, but its use is limited by dose-dependent cardiotoxicity. We previously reported that genetic deletion of p38δ protects female...

Hot off the press from the RTI: RNAi-mediated p38δ silencing mitigates anthracycline cardiotoxicity in female mice
pubmed.ncbi.nlm.nih.gov/41874387/?ut... #RNA #RNATherapeutics

A look back at CHDI’s HD Therapeutics Conference, where our CSO Nicole Datson, PhD, CMO Scott Schobel, MD, and Associate Director Lodewijk Toonen presented data on VO659, our allele-preferential ASO targeting CAG repeat expansion in polyQ diseases.
#HuntingtonsDisease #RNAtherapeutics

Hot off the press from the RTI: An siRNA targeting S6k1 identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration

buff.ly/wHy3jKw #RNA #RNATherapeutics @UMassChan

An siRNA targeting S6k1 identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration - PubMed Age-related macular degeneration (AMD) remains a leading cause for visual impairment in the elderly. We recently showed that activated mammalian target of rapamycin complex 1 (mTORC1) in photoreceptor...

Hot off the press from the RTI: An siRNA targeting <em>S6k1</em> identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration
pubmed.ncbi.nlm.nih.gov/41858838/?ut... #RNA #RNATherapeutics

RTI's Jon Watts speaking at OPT today on Prime Editing with Chemically Modified Templates and Modification-Tolerant Polymerases #RNATherapeutics @JWattsgroup.bsky.social

Hot off the press from the RTI: Lowering the HTT1a transcript as an effective therapy for Huntington's disease in a knockin mouse model

buff.ly/ouOfgTU #RNA #RNATherapeutics @UMassChan

Lowering the HTT1a transcript as an effective therapy for Huntington's disease in a knockin mouse model - PubMed Lowering <i>huntingtin (HTT)</i> transcript levels has been a major focus of therapeutic development for Huntington's disease (HD), but which transcript should be lowered? HD is caused by a CAG repeat...

Hot off the press from the RTI: Lowering the <em>HTT1a</em> transcript as an effective therapy for Huntington's disease in a knockin mouse model
pubmed.ncbi.nlm.nih.gov/41849583/?ut... #RNA #RNATherapeutics

Our CEO Poul Sørensen shared our latest progress this morning at RNA Leaders in Vienna
#RNATherapeutics #Pseudoexon #Parkinsons #Huntingtons #RNAleaders

In extracto cryo-EM reveals eEF2 as a major hibernation factor on 60S and 80S particles Cryogenic electron microscopy (cryo-EM) made impressive progress in resolving cellular macromolecules and their detailed interactions. Single-particle cryo-EM traditionally relies on purified macromolecules...

🧬 New BioRxiv preprint from the Grigorieff & Korostelev labs: In extracto cryo-EM reveals eEF2 as a major hibernation factor on 60S and 80S particles buff.ly/zx8ocVl #RNA #RNATherapeutics @umasschan.bsky.social

Comparative RNA Structure Analysis of Nascent and Mature Transcripts in Saccharomyces cerevisiae - PubMed During transcription, nascent RNA starts base pairing as it exits RNA polymerases (Pols). This base pairing permits the formation of RNA structures that critically influence gene expression at the…

Hot off the press from the Schärfen lab: Comparative RNA Structure Analysis of Nascent and Mature Transcripts in Saccharomyces cerevisiae

buff.ly/pFsKYb9 #RNA #RNATherapeutics @UMassChan.bsky.social

📄 From our special issue on RNA therapeutics:
A study examining cardiac outcomes in DMD treated with the exon-skipping therapy eteplirsen.

Treated patients showed slower LVEF decline and lower risk of reaching cardiac dysfunction thresholds. buff.ly/ycbsqVl

#DMD #RNATherapeutics #ExonSkipping

Hot off the press from the RTI: Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study

buff.ly/AMsTrGa #RNA #RNATherapeutics @UMassChan

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study - PubMed Exon skipping antisense oligonucleotides (AONs) have been extensively studied as a promising method of treating Duchenne muscular dystrophy (DMD), yet the clinical efficacy of the conditionally approved...

Hot off the press from the RTI: Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study
pubmed.ncbi.nlm.nih.gov/41814159/?ut... #RNA #RNATherapeutics

Emw1/TTC27 is a chaperone required for folding of the eukaryotic elongation factor 2 - PubMed Emw1/TTC27 is a chaperone required for folding of the eukaryotic elongation factor 2

Hot off the press from the RTI: Emw1/TTC27 is a chaperone required for folding of the eukaryotic elongation factor 2
pubmed.ncbi.nlm.nih.gov/41806041/?ut... #RNA #RNATherapeutics

Organyl 5'-Phosphates in siRNA Guide Strands: Structure-Function Relationships Governing Anchoring in Argonaute 2 and Metabolic Stability - PubMed Efficient siRNA loading into Argonaute2 (AGO2) requires a 5'-phosphate (5'-P) on the guide strand, yet this group is vulnerable to metabolic degradation in vivo. Although numerous chemical mimics of 5'-P...

Hot off the press from the RTI: Organyl 5'-Phosphates in siRNA Guide Strands: Structure-Function Relationships Governing Anchoring in Argonaute 2 and Metabolic Stability
pubmed.ncbi.nlm.nih.gov/41726981/?ut... #RNA #RNATherapeutics

TAV2b Peptide Derivatives Underwind and Stabilize Double-Stranded RNA upon Binding Double-stranded RNA (dsRNA) has become an essential tool to understand biological processes with promising therapeutic implications. However, its usage is often limited due to poor cellular uptake and...

Recent article from the lab published in @jacs.acspublications.org. Big congrats to first author Zainab Rashid for leading the study and great collaboration with @tomngrossmann.bsky.social and Jocelyne Vreede labs!😃
doi.org/10.1021/jacs...
#singlemolecule #dsRNA #RNAtherapeutics

Divalent siRNA for prion disease Prion protein (PrP) lowering is effective in animal models of prion disease and is being tested clinically in prion disease patients, but there remains a need for more potent PrP-lowering drug candidates....

🧬 Learn more about divalent #siRNAs for prion disease from the Khvorova, Vallabh, & Minikel labs: buff.ly/pdhVPk8
siRNA from bench to clinic: buff.ly/f3i8poM
#RNA #RNATherapeutics @umasschan.bsky.social