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RNA Therapeutics Institute
@rti-umasschan.bsky.social
1.2K followers 210 following 150 posts
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
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RNA Therapeutics Institute @rti-umasschan.bsky.social · Apr 7
The foundation Else Kröner-Fresenius-Stiftung is honoring Dr. Anastasia Khvorova's pioneering work in the field of RNA-based therapies. At 2.5 million euros, the Else Kröner Fresenius Prize for Medical Research is one of the biggest research prizes in the world! www.umassmed.edu/news/news-ar...
rti-umasschan.bsky.social
Hot off the press from the RTI: Dimeric gold nanoparticles enable multiplexed labeling in cryoelectron tomography
buff.ly/4mq5ii3 #RNA #RNATherapeutics
Dimeric gold nanoparticles enable multiplexed labeling in cryoelectron tomography - PubMed
Cryoelectron tomography (cryo-ET) enables three-dimensional visualization of molecular structures within tissue and intact cells, providing a powerful tool for studying the spatial organization of biological...
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November 25, 2025 at 6:02 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: From nematode to Nobel: How community-shared resources fueled the rise of Caenorhabditis elegans as a research organism
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From nematode to Nobel: How community-shared resources fueled the rise of Caenorhabditis elegans as a research organism - PubMed
Experimental organisms such as the nematode <i>Caenorhabditis elegans</i> are fundamental to biological discovery. The success of <i>C. elegans</i> research has been greatly enabled by infrastructure...
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November 25, 2025 at 6:00 PM
rti-umasschan.bsky.social
HAPPENING TODAY AT 5:00PM ET! Sign up for the Basic Biomedical Sciences Umbrella Pathway Admissions Workshop on Tues, Nov 18, 2025: direc.to/nA7S

Attend our admissions workshop to get your questions answered ahead of the Dec 1, 2025 deadline for graduate applications.
November 18, 2025 at 6:46 PM
rti-umasschan.bsky.social
We are so excited for inaugural Yongliang Liao Distinguished Lecture, given by Dr. Rachel Green, Johns Hopkins University on ZAK activation at the colliding ribosome! We are grateful for Dr. Amy Liao and her family for making this lecture possible!
November 17, 2025 at 8:16 PM
rti-umasschan.bsky.social
Join a collaborative home of Nobel Prize-winning RNA research! The Morningside Graduate School of Biomedical Sciences is accepting applications for the new RNA Therapeutics & Biology Track in the Biomedical Sciences PhD program: direc.to/nEHU
#PhDChat #Gradschool #PhDSky
Biomedical Sciences PhD | UMass Chan Medical School
Advance the treatment and prevention of disease by earning your Biomedical Sciences PhD at the UMass Chan Medical School. Learn more.
direc.to
November 16, 2025 at 3:00 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding
buff.ly/KZpiqXh #RNA #RNATherapeutics
Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding - PubMed
Huntington's disease arises from a CAG expansion in the huntingtin gene beyond a critical threshold. Current therapeutics primarily aim to reduce toxicity by lowering levels of mutant HTT mRNA and protein....
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November 15, 2025 at 6:00 PM
rti-umasschan.bsky.social
OPEN FOR GRAD APPLICATIONS!! The NEW RNA Therapeutics & Biology Program in the Basic Biomedical Sciences PhD program develops leaders. Register for the workshop to get qs answered ahead of Dec 1, 2025 app deadline.
Admission Workshop Tues, Nov 18, 2025: direc.to/nA7S
At UMass Chan, where Nobel Laureates pioneered small RNA discoveries, we empower the next RNA GIANTS
November 14, 2025 at 5:00 PM
rti-umasschan.bsky.social
RTI @UMassChan scientists have received a $6M+ grant to evaluate gene editing technologies for treating frontotemporal dementia: direc.to/nE19
#FTD #RNATx
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UMass Chan Medical School scientists to assess gene editing technologies for treating frontotemporal dementia
UMass Chan scientists have received funding to explore gene editing technologies for treating frontotemporal dementia.
www.umassmed.edu
November 14, 2025 at 3:39 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD
buff.ly/X23QYQI #RNA #RNATherapeutics
TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD - PubMed
Motor neuron hyperexcitability is a broadly observed yet poorly understood feature of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Nuclear depletion and cytoplasmic aggregation...
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November 2, 2025 at 6:02 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Phage SSB detection by retron Eco8 msDNA unleashes nuclease-mediated immunity
buff.ly/HXnSgjd #RNA #RNATherapeutics
Phage SSB detection by retron Eco8 msDNA unleashes nuclease-mediated immunity - PubMed
Retrons are antiphage defense systems that synthesize multicopy single-stranded DNA (msDNA) and are being adapted for genome engineering. The Escherichia coli retron Eco8 system comprises a reverse transcriptase...
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November 2, 2025 at 6:00 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Structural mechanism of mRNA decoding by mammalian GTPase GTPBP1
buff.ly/oYbdavv #RNA #RNATherapeutics
Structural mechanism of mRNA decoding by mammalian GTPase GTPBP1 - PubMed
GTP-binding protein 1 (GTPBP1) is a widespread translational GTPase closely related to elongation factor eEF1A. The loss of GTPBP1 leads to errors in neuronal development in animals and is associated...
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October 14, 2025 at 5:04 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: mRNA initiation and termination are spatially coordinated
buff.ly/vwSuPWC #RNA #RNATherapeutics
mRNA initiation and termination are spatially coordinated - PubMed
Transcriptional initiation and termination decisions drive messenger RNA (mRNA) isoform diversity but the relationship between them remains poorly understood. By systematically profiling joint usage of...
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October 10, 2025 at 5:02 PM
rti-umasschan.bsky.social
Hot off the press from the @korostelevlab.bsky.social Structural mechanism of mRNA decoding by mammalian GTPase GTPBP1

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September 29, 2025 at 2:33 PM
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Hot off the press from the RTI: Defining the roles of the Integrator, NEXT, and nuclear exosome complexes in Drosophila oogenesis
buff.ly/cWIXcBu #RNA #RNATherapeutics
Defining the roles of the Integrator, NEXT, and nuclear exosome complexes in Drosophila oogenesis - PubMed
Nuclear RNA homeostasis depends on the balance of transcription, RNA processing, degradation, and transport between the nucleus and cytoplasm. RNA degradation directed by the Integrator, nuclear exosome...
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September 25, 2025 at 5:01 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Cryopreservation of siRNA-Treated Cells Is Feasible
buff.ly/BWdy50S #RNA #RNATherapeutics
Cryopreservation of siRNA-Treated Cells Is Feasible - PubMed
Cryopreservation is a routine step in the manufacturing process of adoptive cell therapies (ACT), providing critical logistic flexibility. RNA interference (RNAi)-based therapies are increasingly being...
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September 25, 2025 at 5:01 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: New insights into the role of IFN-α/β and TLR7/8/9 in cancer immunotherapy and systemic autoimmunity
buff.ly/SuU8jSW #RNA #RNATherapeutics
New insights into the role of IFN-α/β and TLR7/8/9 in cancer immunotherapy and systemic autoimmunity - PubMed
The role of type I interferon (IFN) in mediating tumor immunosurveillance, in situ vaccination, and response to other cancer immunotherapies is well established. The biologic and species-specific differences...
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September 20, 2025 at 5:00 PM
Reposted by RNA Therapeutics Institute
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UMass Chan licenses exNA oligonucleotide technology to Hongene Biotech Corporation: direc.to/nu79

By making exNA phosphoramidites and oligonucleotides available to researchers, Hongene hopes to advance more stable, tissue-specific RNA therapeutics. ‪

#exNA #RNA @rti-umasschan.bsky.social
UMass Chan Medical School licenses exNA oligonucleotide technology to Hongene Biotech Corporation
UMass Chan Medical licenses exNA oligonucleotide technology to Hongene Biotech Corporation
direc.to
September 3, 2025 at 1:23 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: A single amino acid variant in the variable region I of AAV capsid confers liver detargeting
buff.ly/nmjkDIT #RNA #RNATherapeutics
A single amino acid variant in the variable region I of AAV capsid confers liver detargeting - PubMed
AAV capsid serotypes isolated from nature have been widely used in gene delivery and gene therapy. Recently, more than 1,000 distinct AAV capsids were identified from human clinical samples by high-throughput,...
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September 18, 2025 at 5:01 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation
buff.ly/oMspoFO #RNA #RNATherapeutics
Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation - PubMed
<span><i>HTT1a</i> was identified in human and mouse Huntington's disease brain as the pathogenic exon 1 mRNA generated from aberrant splicing between exon 1 and 2 of <i>HTT</i> that contributes to aggregate...
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September 11, 2025 at 5:02 PM
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It's back to school time in the RTI!!! Come join the Xue Lab for a Brainstorm “Classroom” Challenge with snacks and cake to celebrate another great school year! TODAY at 4pm in the 4th floor lobby!
September 4, 2025 at 4:05 PM
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ICYMI: Newest article from the Xue, Flotte, & Sontheimer labs: A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
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A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is s…
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September 3, 2025 at 9:47 PM
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Hot off the press from the RTI: Translon: a single term for translated regions
pubmed.ncbi.nlm.nih.gov/40890551/?ut... #RNA #RNATherapeutics
Translon: a single term for translated regions - PubMed
Translon: a single term for translated regions
pubmed.ncbi.nlm.nih.gov
September 2, 2025 at 5:02 PM
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Hot off the press from the RTI: A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
pubmed.ncbi.nlm.nih.gov/40883986/?ut... #RNA #RNATherapeutics
A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models - PubMed
Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is sequestered in hepatocytes, causing lung emphysema...
pubmed.ncbi.nlm.nih.gov
August 31, 2025 at 5:00 PM
rti-umasschan.bsky.social
Hot off the press from the RTI: Pervasive noise in human splice site selection
pubmed.ncbi.nlm.nih.gov/40791517/?ut... #RNA #RNATherapeutics
Pervasive noise in human splice site selection - PubMed
RNA splicing has historically been thought to be highly efficient and accurate, with little opportunity for deviation from regulated alternative splicing decisions. This dogma has been challenged by recent...
pubmed.ncbi.nlm.nih.gov
August 26, 2025 at 5:03 PM
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Hot off the press from the RTI: Therapeutic RNA cleavage with RfxCas13d in Hutchinson-Gilford progeria syndrome
pubmed.ncbi.nlm.nih.gov/40829594/?ut... #RNA #RNATherapeutics
Therapeutic RNA cleavage with RfxCas13d in Hutchinson-Gilford progeria syndrome - PubMed
Therapeutic RNA cleavage with RfxCas13d in Hutchinson-Gilford progeria syndrome
pubmed.ncbi.nlm.nih.gov
August 20, 2025 at 5:01 PM