Hot off the press from the RTI: Systematic identification of germ granule proteins reveals specialized roles in RNAi and small RNA inheritance buff.ly/qZJWqsB #RNA #RNATherapeutics @UMassChan
RNA Therapeutics Institute
@rti-umasschan.bsky.social
The RNA Therapeutics Institute (RTI) at UMass Chan Medical School. Uniting nucleic acid scientists w/clinicians to devise human therapies. Loving RNA since before it was cool. #RNATx2025
Hot off the press from the RTI: Systematic identification of germ granule proteins reveals specialized roles in RNAi and small RNA inheritance pubmed.ncbi.nlm.nih.gov/42538287/?ut... #RNA #RNATherapeutics
Systematic identification of germ granule proteins reveals specialized roles in RNAi and small RNA inheritance - PubMed
Biomolecular condensates, such as germ granules, organize RNAi pathways critical for fertility and genome regulation. However, the protein composition and functional contributions of these condensates...
pubmed.ncbi.nlm.nih.gov
Hot off the press from the RTI: PPMO therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein buff.ly/g2Mrvy0 #RNA #RNATherapeutics @UMassChan
Hot off the press from the RTI: PPMO therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein pubmed.ncbi.nlm.nih.gov/42446943/?ut... #RNA #RNATherapeutics
PPMO therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein - PubMed
The dysferlinopathies are a spectrum of autosomal recessive muscle diseases caused by mutations in the dysferlin gene (DYSF) gene. Clinical manifestations vary from asymptomatic hyperCKemia to severe...
pubmed.ncbi.nlm.nih.gov
Hot off the press from the RTI: Precision RNAi for Fibrodysplasia Ossificans Progressiva: a combinatorial, unimolecular, allele selective approach buff.ly/O9ffiCP #RNA #RNATherapeutics @UMassChan
Hot off the press from the RTI: Precision RNAi for Fibrodysplasia Ossificans Progressiva: a combinatorial, unimolecular, allele selective approach pubmed.ncbi.nlm.nih.gov/42370290/?ut... #RNA #RNATherapeutics
Precision RNAi for Fibrodysplasia Ossificans Progressiva: a combinatorial, unimolecular, allele selective approach - PubMed
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder caused by a dominant mutation in the <i>ACVR1</i> gene (R206H, 97% of cases), leading to debilitating heterotopic ossification (HO)...
pubmed.ncbi.nlm.nih.gov
Hot off the press from the RTI: G quadruplex DNA facilitates a pervasive path to homologous recombination pubmed.ncbi.nlm.nih.gov/42282655/?ut... #RNA #RNATherapeutics
G quadruplex DNA facilitates a pervasive path to homologous recombination - PubMed
Homologous recombination (HR) requires efficient homology search and strand invasion, yet how homologous templates are identified within nuclear space remains unclear. Here, we identify G-quadruplex (G4)...
pubmed.ncbi.nlm.nih.gov
🧬 New BioRxiv preprint from the RTI: Improved cryo-EM reconstruction of sub-50 kDa complexes using 2D template matching buff.ly/nEKQ1rN #RNA #RNATherapeutics @umasschan.bsky.social
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Hot off the press from the RTI: Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo pubmed.ncbi.nlm.nih.gov/42244699/?ut... #RNA #RNATherapeutics
Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo - PubMed
Precision engineering of CRISPR/Cas9 components has advanced genome editing toward therapeutic applications. Completely chemically stabilized guide RNAs (gRNAs) have the potential to improve <i>in vivo</i>...
pubmed.ncbi.nlm.nih.gov
THANK YOU to all our speakers, panelists, attendees and sponsors for another successful #RNATx2026! Save the dates and join us next year at #RNATx2027 June 23-25, 2027!!
Riccardo Pannella, From Target Discovery to Clinical Translation: The Development of RES-010 #RNATx2026
Translational Discovery session up next! T. Scott Johnson discussing Cross-Species Metabolism & PK of CBP-4888 Including SC & IV Administration in Healthy Individuals #RNATx2026 #siRNA #preeclampsia
Ailong Ke discussing Less is more: Conversion of IscB and Cas9 into RNA-Guided RNA Editors #RNATx2026
Ailong Ke discussing Less is more: Conversion of IscB and Cas9 into RNA-Guided RNA Editors #RNATx2026
Editing session kicks off with Sam Sternberg discussing RNA-Guided DNA Integration for Therapeutic Genome and Microbiome Engineering #RNATx2026
Truly fantastic talk of biotech, business, culture, strategy, and a beautiful historical perspective of the RNAi from concept to clinic from John Maraganore. #RNATx2026 @JMaraganore
We kick off Day 3 with biotech leader, founding CEO of Alnylam, and one of RTI’s ardent supporters, John Maraganore! Also a fantastic moment for John and our inaugural Maraganore fellow Leo Schärfen! @JMaraganore @LeoSchaerf #RNATx2026
🧬 New BioRxiv preprint from the RTI: Mismatch repair dissection by in vivo RNAi reveals dose-dependent modulators of somatic instability and proteome remodeling in Huntingtons disease buff.ly/2PmGcMj #RNA #RNATherapeutics @umasschan.bsky.social
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Sophia Lugo on RADAR: RNA Sense-and-Response Systems for Precision Targeting of Genetic Payloads #RNATx2026
Sun Hur discussing Innate Immune Mechanism for Sensing Self vs Non-self RNA Discrimination #RNATx2026
The RTI’s chair, Dr. Phil Zamore, is speaking on Muscle-specific RNA Silencing by #miRNA Tether #RNATx2026
Silvi Rouskin speaking at #RNATx2026 on Conformational Ensembles Reveal a Missing Layer of pri-miRNA Regulation
Next at #RNATx2026 Anna Marie Pyle on Discovery and Targeting of RNA Regulatory Structures in Viral Pathogens
Next up Pedro Miura on Leveraging Long Reads to Understand RNA Processing in Neural Development #RNATX2026
Kicking off Day 2 of #RNATx2026 with Karmella Haynes on Synthetic #lncRNAs Reveal Design Constraints on RNA-Mediated Chromatin Silencing #ncRNA #epigenetics
#RNATx2026 has kicked off, first with an introduction from co-organizer Phil Zamore and now our first keynote, Xiaowei Zhuang with some gorgeous MERFISH imaging!
Hot off the press from the RTI: Antisense oligonucleotides targeting Ninjurin1 ameliorate the pathology of lead- and cadmium-induced chronic obstructive pulmonary disease in mice buff.ly/A9VP5xu #RNA #RNATherapeutics @UMassChan
Hot off the press from the RTI: Antisense oligonucleotides targeting Ninjurin1 ameliorate the pathology of lead- and cadmium-induced chronic obstructive pulmonary disease in mice pubmed.ncbi.nlm.nih.gov/42337738/?ut... #RNA #RNATherapeutics
Antisense oligonucleotides targeting Ninjurin1 ameliorate the pathology of lead- and cadmium-induced chronic obstructive pulmonary disease in mice - PubMed
These findings establish Ninj1 as a critical mediator of chronic lung inflammation and a translationally relevant therapeutic target, highlighting Ninj1-directed ASO therapy as a promising strategy for...
pubmed.ncbi.nlm.nih.gov