A multidisciplinary UMass Chan research team has received $14.7 million to develop new models and gene therapies for alpha-1 antitrypsin deficiency, a genetic defect which causes severe loss of lung function: https://bit.ly/4cEbJj7 #GeneTherapy @xueumass.bsky.social @rti-umasschan.bsky.social

UMass Chan receives $14.7 million grant to develop gene therapies for alpha-1 antitrypsin deficiency

Scientists receive $14.7 million to improve vector design for gene therapies targeting alpha-1 antitrypsin deficiency

umassmed.edu

Welcome back from the summer, we hope you're ready for the new school year! See all the updates in the RTI Messenger: buff.ly/DzhfV9X

https://go.umassmed.edu/webmail/184882/6558196230/02b5b8a899d153681bea898e35437478d2d67de2ef91a34c040ddf370fdeab94

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Hot off the press from the RTI: In extracto cryo-EM reveals eEF2 as a major hibernation factor on 60S and 80S particles buff.ly/GNsNOdR #RNA #RNATherapeutics @UMassChan

Hot off the press from the RTI: Early reduction of myelin-associated glycoprotein at myelin membranes in Huntington's disease buff.ly/IjpxT9a #RNA #RNATherapeutics @UMassChan

Short interfering RNAs (siRNAs) are on Peter Parker’s mind in the newest Spider-Man movie. Anastasia Khvorova, PhD, of UMass Chan Medical School, spoke with @sciencenews.bsky.social about #siRNAs and what the movie got right (and wrong): https://direc.to/pn9y #SpiderMan @rti-umasschan.bsky.social

What Spider-Man gets right — and wrong — about turning off superpowers

In 'Spider-Man: Brand New Day,' Peter Parker devises a superpower inhibitor based on a real therapy using RNA.

direc.to

Erik J. Sontheimer, PhD,  a pioneering leader in the field of CRISPR gene editing, will become the next chair of the RNA Therapeutics Institute (RTI) in January. Read about Dr. Sontheimer and the RTI here: https://direc.to/pn_q @eriksontheimer.bsky.social @rti-umasschan.bsky.social

Erik Sontheimer named chair of RNA Therapeutics Institute at UMass Chan, effective January 2027

Erik J. Sontheimer, PhD, the Pillar Chair in Biomedical Research, professor and vice chair of RNA therapeutics, has been appointed chair of the RNA Therapeutics Institute, effective in January of 2027...

direc.to

Hot off the press from the RTI: Cross-species comparative spatial transcriptomics of hair follicle-T cell interactions identifies conserved drivers of cutaneous lupus erythematosus skin disease and associated hair loss buff.ly/CV44nLR #RNA #RNATherapeutics @UMassChan

Congrats to the Awardees of the 2026 Michael R. Green, MD, PhD, Award in Graduate Research, Andrew Petti (RTI; Wen Xue's Lab) and Brennon Berard (MCCB; Dohoon Kim's Lab). Save the Date for their research presentations: Tuesday Sept 8, 2026 at 11:00am DiMare Center, N1-1500 #generegulation #RNATx

'Shutting down foreign DNA — such as inserted arachnid DNA — or mutated genes is the sort of thing #siRNAs do well, says Anastasia Khvorova, a chemical biologist at the UMass Chan Medical School’s #RNA Therapeutics Institute. Read more about the RTI & #SpiderMan 🕸️ buff.ly/OqHDgb7

Hot off the press from the RTI: Improved Purification of sgRNA and pegRNA by Use of a 5'-Terminal Dioctyloxytrityl (DOT) Protecting Group buff.ly/meigSEe #RNA #RNATherapeutics @UMassChan

Hot off the press from the RTI: Improved Purification of sgRNA and pegRNA by Use of a 5'-Terminal Dioctyloxytrityl (DOT) Protecting Group pubmed.ncbi.nlm.nih.gov/42631961/?ut... #RNA #RNATherapeutics

Hot off the press from the RTI: Pervasive noise in human pre-mRNA splice site selection buff.ly/coo64aa #RNA #RNATherapeutics @UMassChan

Hot off the press from the RTI: Pervasive noise in human pre-mRNA splice site selection pubmed.ncbi.nlm.nih.gov/42607686/?ut... #RNA #RNATherapeutics

First mRNA flu shot approved by FDA bodes well for improving drugs of the future – though a few hurdles remain before #mRNA can move beyond vaccines buff.ly/2jVCuh7

First mRNA flu shot approved by FDA bodes well for improving drugs of the future – though a few hurdles remain before mRNA can move beyond vaccines

Li Li, PhD, discusses how the FDA's approval of the first mRNA flu vaccine could accelerate the development of future mRNA-based therapies while highlighting the scientific challenges that remain.

umassmed.edu

Hot off the press from the RTI: Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington's disease pathogenesis in mice buff.ly/MK0i321 #RNA #RNATherapeutics @UMassChan

Hot off the press from the RTI: Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington's disease pathogenesis in mice pubmed.ncbi.nlm.nih.gov/42585290/?ut... #RNA #RNATherapeutics

Hot off the press from the RTI: Viral gene replication enhances AAV vector quality and reduces manufacturing costs buff.ly/LnqZQ8S #RNA #RNATherapeutics @UMassChan

Hot off the press from the RTI: Viral gene replication enhances AAV vector quality and reduces manufacturing costs pubmed.ncbi.nlm.nih.gov/42581546/?ut... #RNA #RNATherapeutics

Viral gene replication enhances AAV vector quality and reduces manufacturing costs - PubMed

Viral vectors for gene and cell therapy are typically produced by transfecting plasmids into mammalian cells. Because plasmid DNA is not replicated in these cells, large quantities are required to express...

pubmed.ncbi.nlm.nih.gov

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

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

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