Lightnews — Scholar-powered news

RNA Therapeutics Institute @rti-umasschan.bsky.social · 9d

Hot off the press from the @korostelevlab.bsky.social Structural mechanism of mRNA decoding by mammalian GTPase GTPBP1

buff.ly/MXMCnRZ

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · 13d

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...

buff.ly

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · 13d

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...

buff.ly

RNA Therapeutics Institute @rti-umasschan.bsky.social · 18d

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...

buff.ly

1

Reposted by RNA Therapeutics Institute

UMass Chan Medical School @umasschan.bsky.social · Sep 3

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

1 3

RNA Therapeutics Institute @rti-umasschan.bsky.social · 20d

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,...

buff.ly

RNA Therapeutics Institute @rti-umasschan.bsky.social · 27d

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

HTT1a 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 HTT that contributes to aggregate...

buff.ly

RNA Therapeutics Institute @rti-umasschan.bsky.social · Sep 4

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!

RNA Therapeutics Institute @rti-umasschan.bsky.social · Sep 3

ICYMI: Newest article from the Xue, Flotte, & Sontheimer labs: A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models
buff.ly/OA3OCXz

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…

buff.ly

RNA Therapeutics Institute @rti-umasschan.bsky.social · Sep 2

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

2

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 31

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

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 26

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

1 3

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 20

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

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 17

Hot off the press from the RTI: RNA Pol II inhibition activates cell death independently from the loss of transcription
pubmed.ncbi.nlm.nih.gov/40818455/?ut... #RNA #RNATherapeutics

RNA Pol II inhibition activates cell death independently from the loss of transcription - PubMed

RNA Pol II-mediated transcription is essential for eukaryotic life. Although loss of transcription is thought to be universally lethal, the associated mechanisms promoting cell death are not yet known....

pubmed.ncbi.nlm.nih.gov

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 15

Hot off the press from the RTI: Optimized Chemical Structure Extends Silencing Duration of Therapeutic siRNAs in Dividing Cancer and Immune Cells
pubmed.ncbi.nlm.nih.gov/40808258/?ut... #RNA #RNATherapeutics

Optimized Chemical Structure Extends Silencing Duration of Therapeutic siRNAs in Dividing Cancer and Immune Cells - PubMed

RNA interference (RNAi)-based medicines offer precise targeting of virtually any transcript, making them an appealing new drug class for immune-oncology. While RNAi therapies are highly effective in non-dividing...

pubmed.ncbi.nlm.nih.gov

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 13

Hot off the press from the RTI: Epigenetic control of topoisomerase 1 activity presents a cancer vulnerability
pubmed.ncbi.nlm.nih.gov/40796804/?ut... #RNA #RNATherapeutics

Epigenetic control of topoisomerase 1 activity presents a cancer vulnerability - PubMed

DNA transactions introduce torsional constraints that pose an inherent risk to genome integrity. While topoisomerase 1 (TOP1) activity is essential for DNA supercoil removal, the aberrant stabilization...

pubmed.ncbi.nlm.nih.gov

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 11

RTI shared how science is being affected by disastrous NIH funding cuts. Basic science is imperative to developing therapeutics; NIH funding contributed to research associated with almost every new drug (99.4%) approved by FDA between 2010 & 2019.
www.washingtonpost.com/business/202...

www.washingtonpost.com

1

RNA Therapeutics Institute @rti-umasschan.bsky.social · Aug 1

Hot off the press from the RTI: Prime assembly with linear DNA donors enables large genomic insertions
pubmed.ncbi.nlm.nih.gov/40667017/?ut... #RNA #RNATherapeutics

Prime assembly with linear DNA donors enables large genomic insertions - PubMed

Targeted insertion of large DNA fragments has promising applications for genome engineering and gene therapy1,2. Twin prime editing (PE) guide RNAs (pegRNAs) have enabled relatively large insertions,...

pubmed.ncbi.nlm.nih.gov

1 1

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 30

Hot off the press from the RTI: Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice
pubmed.ncbi.nlm.nih.gov/40667291/?ut... #RNA #RNATherapeutics

Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice - PubMed

Huntington's disease (HD) is a progressive neurodegenerative disorder with no approved therapies. Two major molecular drivers-somatic expansion of inherited CAG repeats and toxic mutant HTT (mHTT) variants-lead...

pubmed.ncbi.nlm.nih.gov

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 29

Hot off the press from the RTI: Swine reporter model for preclinical evaluation and characterization of gene delivery vectors
pubmed.ncbi.nlm.nih.gov/40661506/?ut... #RNA #RNATherapeutics

Swine reporter model for preclinical evaluation and characterization of gene delivery vectors - PubMed

Delivery of gene therapy vectors targeted to any somatic cell remains a key barrier for the development of genetic medicines. While rodent models provide insights into vector biodistribution and cellular...

pubmed.ncbi.nlm.nih.gov

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 26

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/40654892/?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

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 22

We couldn't be more excited for @leoshaerfen.bsky.social to be joining us as the RTI's inaugural Maraganore Fellow!!

Leo Schärfen @leoschaerfen.bsky.social · Jul 22

Fun news: I'm starting my research group at @rti-umasschan.bsky.social as the inaugural Maraganore Fellow! Our lab will focus on the RNA sequence-structure-function relationship in the cellular environment.

Visit our website to explore open positions: schaerfenlab.com

#RNA #Hiring #PostbacJobs

1

Reposted by RNA Therapeutics Institute

Leo Schärfen @leoschaerfen.bsky.social · Jul 22

Fun news: I'm starting my research group at @rti-umasschan.bsky.social as the inaugural Maraganore Fellow! Our lab will focus on the RNA sequence-structure-function relationship in the cellular environment.

Visit our website to explore open positions: schaerfenlab.com

#RNA #Hiring #PostbacJobs

1 3 10

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 18

Hot off the press from the RTI: Localization of Ribosomal DNA-Associated Retrotransposons Refines the Heterochromatin Map of the X Chromosome in Drosophila melanogaster
pubmed.ncbi.nlm.nih.gov/40505636/?ut... #RNA #RNATherapeutics

Localization of Ribosomal DNA-Associated Retrotransposons Refines the Heterochromatin Map of the X Chromosome in Drosophila melanogaster - PubMed

We identified distinct clusters of both R1 and R2 elements within the X chromosome heterochromatin outside the NOR. R1 elements are highly enriched in the region between the heterochromatic Stellate (hSte)...

pubmed.ncbi.nlm.nih.gov

RNA Therapeutics Institute @rti-umasschan.bsky.social · Jul 18

Hot off the press from the RTI: A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic
pubmed.ncbi.nlm.nih.gov/40673060/?ut... #RNA #RNATherapeutics

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic - PubMed

Facioscapulohumeral muscular dystrophy (FSHD) is the third most diagnosed muscular dystrophy. The disease is caused by genetic and epigenetic disruptions that result in misexpression of the germline transcription...

pubmed.ncbi.nlm.nih.gov