2018 News
December 19, 2018, New publication from the Aronin and Khvorova labs. Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease. Tousley A, Iuliano M, Weisman E, Sapp E, Zhang N, Vodicka P, Alexander J, Aviolat H, Gatune L, Reeves P, Li X, Khvorova A, Ellerby ML, Aronin N, DiFiglia M, Kegel-Gleason BK. J Huntingtons Dis. 2018 Dec 28. doi: 10.3233/JHD-180311. [Epub ahead of print] PMID: 30594931
December 18, 2018 - New publication from the Mello Lab. A C. elegans Model for the Rare Human Channelopathy, Timothy Syndrome Type 1. Lagoy R, Kim H, Mello C, Albrecht D. MicroPubl Biol. 2018 Dec 18;2018. doi: 10.17912/micropub.biology.000081. No abstract available. PMID: 32550369
December 18, 2018, New publication from the Sontheimer lab. A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing. Edraki A, Mir A, Ibraheim R, Gainetdinov I, Yoon Y, Song CQ, Cao Y, Gallant J, Xue W, Rivera-Pérez JA, Sontheimer EJ. Mol Cell. 2018 Dec 18. pii: S1097-2765(18)31033-5. doi: 10.1016/j.molcel.2018.12.003. [Epub ahead of print] PMID: 30581144
December 18, 2018, New publication from the Khvorova lab. Gene Silencing with siRNA (RNA Interference): A New Therapeutic Option during Ex-vivo Machine Liver Perfusion Preservation. Thijssen MF, Brüggenwirth IMA, Gillooly A, Khvorova A, Kowalik TF, Martins PN.Liver Transpl. 2018 Dec 18. doi: 10.1002/lt.25383. [Epub ahead of print]PMID: 30561891
December 15, 2018, New publication from the Grunwald lab. Impact of optical aberrations on axial position determination by photometry. Thorsen RØ, Hulleman CN, Hammer M, Grünwald D, Stallinga S, Rieger B. Nat Methods. 2018 Dec;15(12):989-990. doi: 10.1038/s41592-018-0227-4. PMID: 30504865
December 14, 2018, New publication from the Khvorova lab. Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo. Biscans A, Coles A, Haraszti R, Echeverria D, Hassler M, Osborn M, Khvorova A. Nucleic Acids Res. 2018 Dec 14. doi: 10.1093/nar/gky1239. [Epub ahead of print] PMID: 30544191
December 8, 2018, New publication from the Khvorova lab. Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways. Osborn MF, Coles AH, Biscans A, Haraszti RA, Roux L, Davis S, Ly S, Echeverria D, Hassler MR, Godinho BMDC, Nikan M, Khvorova A.Nucleic Acids Res. 2018 Dec 8. doi: 10.1093/nar/gky1232. [Epub ahead of print] PMID: 30535404
December 5, 2018, New publication from the Grunwald lab. Deconstructing transport-distribution reconstruction in the nuclear-pore complex. Tu LC, Huisman M, Chung YC, Smith CS, Grunwald D. Nat Struct Mol Biol. 2018 Dec 5. doi: 10.1038/s41594-018-0161-2. [Epub ahead of print] PMID: 30518848
December 5, 2018, New publication from the Sontheimer lab. NmeCas9 is an intrinsically high-fidelity genome-editing platform. Amrani N, Gao XD, Liu P, Edraki A, Mir A, Ibraheim R, Gupta A, Sasaki KE, Wu T, Donohoue PD, Settle AH, Lied AM, McGovern K, Fuller CK, Cameron P, Fazzio TG, Zhu LJ, Wolfe SA, Sontheimer EJ. Genome Biol. 2018 Dec 5;19(1):214. doi: 10.1186/s13059-018-1591-1. PMID: 30518407
December 4, 2018, New publication from the Sontheimer lab. Potent Cas9 Inhibition in Bacterial and Human Cells by AcrIIC4 and AcrIIC5 Anti-CRISPR Proteins. Lee J, Mir A, Edraki A, Garcia B, Amrani N, Lou HE, Gainetdinov I, Pawluk A, Ibraheim R, Gao XD, Liu P, Davidson AR, Maxwell KL, Sontheimer EJ.MBio. 2018 Dec 4;9(6). pii: e02321-18. doi: 10.1128/mBio.02321-18. PMID: 30514786
December 4, 2018, New publication from the Sontheimer lab. Adapting dCas9-APEX2 for subnuclear proteomic profiling. Gao XD, Rodríguez TC, Sontheimer E2. Methods Enzymol. 2019;616:365-383. doi: 10.1016/bs.mie.2018.10.030. Epub 2018 Dec 10.
November 19, 2018, New publication from the Khvorova & Moore labs. RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, Zsengeller ZK, Hennessy A, Karumanchi SA, Moore MJ, Khvorova A. Nat Biotechnol. 2018 Nov 19. doi: 10.1038/nbt.4297. [Epub ahead of print]. PMID: 30451990
Press coverage:
- STAT News: New research highlights an unexpected future for RNAi-based drugs — for pregnant women
- UMass Chan Medical School researchers find potential therapy for preeclampsia
- RNAi therapy mitigates preeclampsia symptoms in UMass Chan Medical School study
- RNAi Therapy for Preeclampsia is Pregnant with Possibility
- RNAi therapy mitigates preeclampsia symptoms
November 19, 2018, New publication from the Wolfe and Sontheimer labs. Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing. Bolukbasi MF, Liu P, Luk K, Kwok SF, Gupta A, Amrani N, Sontheimer EJ, Zhu LJ, Wolfe SA. Nat Commun. 2018 Nov 19;9(1):4856. doi: 10.1038/s41467-018-07310-x. PMID: 30451839
November 18, 2018, The RTI's Erik Sontheimer, Anastasia Khvorova, Jon Watts along with Scot Wolfe and the RTI's Wen Xue along with Guanping Gao all join the National Institutes of Health’s Somatic Cell Genome Editing Consortium after each receiving UG3 awards, major collaborative awards. The NIH Common Fund’s Somatic Cell Genome Editing program aims to develop quality tools to perform effective and safe genome editing in human patients.
Press Coverage:
November 16, 2018, New review from the Zamore Lab. PIWI-interacting RNAs: small RNAs with big functions. Ozata DM, Gainetdinov I, Zoch A, O'Carroll D, Zamore PD. Nat Rev Genet. 2018 Nov 16. doi: 10.1038/s41576-018-0073-3. [Epub ahead of print] Review. PMID: 30446728
November 10, 2018, The RTI's Nobel Laureate, Craig Mello, honored in Fall River, MA. The event put on by the Portuguese American community at the Cove restaurant in Fall River.
November 12, 2018, New publication from the Zamore lab. Maelstrom Represses Canonical Polymerase II Transcription within Bi-directional piRNA Clusters in Drosophila melanogaster. Chang TH, Mattei E, Gainetdinov I, Colpan C, Weng Z, Zamore PD. Mol Cell. 2018 Nov 12. pii: S1097-2765(18)30932-8. doi: 10.1016/j.molcel.2018.10.038. [Epub ahead of print] PMID: 30527661
November 8, 2018, New publication from the Dekker & Moore labs. Higher-Order Organization Principles of Pre-translational mRNPs. Mihir Metkar M, Ozadam H, Lajoie BR, Imakaev M, Mirny LA, Dekker J, Moore M. Mol Cell Nov 8, 2018, doi:https://doi.org/10.1016/j.molcel.2018.09.012
November 7, 2018, New publication from Sena-Esteves, Flotte, Aronin & Gao labs. Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer. Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G. Mol Ther Methods Clin Dev. 2018 Oct 4;11:65-72. doi: 10.1016/j.omtm.2018.09.003. eCollection 2018 Dec 14. PMID: 30397628 [PubMed]
October 30, 2018, New publication from the Grunwald & Pederson labs. CRISPR-Sirius: RNA scaffolds for signal amplification in genome imaging. Ma H, Tu LC, Naseri A, Chung YC, Grunwald D, Zhang S, Pederson T. Nat Methods. 2018 Nov;15(11):928-931. doi: 10.1038/s41592-018-0174-0. Epub 2018 Oct 30. PMID: 30377374 [PubMed - in process]
October 26, 2018, RTI Nobel Laureate, Craig Mello, to be honored in Fall River, MA. On Nov. 10, Mello will be recognized during a special brunch and birthday bash for the Portuguese American community at the Cove restaurant in Fall River. The event will take place from 10 am to 2 pm.
October 19, 2018, New publication from the Khvorova & Aronin labs. Exosomes produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity.
Haraszti RA, Miller R, Stoppato M, Sere YY, Coles A, Didiot MC, Wollacott R, Sapp E, Dubuke M, Li X, Shaffer S, DiFiglia M, Wang Y, Aronin N, Khvorova A. Mol Ther. 2018 Sep 22. pii: S1525-0016(18)30456-8. doi: 10.1016/j.ymthe.2018.09.015. [Epub ahead of print]
October 18, 2018, Julia Alterman (Khvorova lab) presented a webinar for the Huntington's Disease Society of America (HDSA). RNAi-based huntingtin lowering in the central nervous system: a new potential HD therapeutic.
October 1, 2018 Congratulations to Julia Alterman, Bruno Godhino, Chantal Ferguson and Anton Turanov, (all from the Khvorova lab) for winning poster prizes at the Oligonucleotide Therapeutics Society annual meeting!
October 1, 2018, Congratulations to Cansu Colpan (Zamore lab), Chantal Ferguson (Khvorova lab), and Zachary Kennedy (Xue lab) for receiving travel awards from the Oligonucleotide Therapeutics Society for the annual meeting!
September 29, 2018, New publication from the Aronin lab. Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. Mondo E, Moser R, Gao G, Mueller C, Sena-Esteves M, Sapp E, Pfister E, O'Connell D, Takle K, Erger KE, Liu W, Conlon TJ, DiFiglia M, Gounis MJ, Aronin N. J Huntingtons Dis. 2018 Sep 29. doi: 10.3233/JHD-180302. [Epub ahead of print]
September 14, 2018, New review from the Pai lab. Environmental influences on RNA processing: Biochemical, molecular and genetic regulators of cellular response. Pai AA, Luca F. WIREs RNA. 14 Sept 2018.
September 13, 2018, New Publication from the Mello lab. Robust Genome Editing With Short Single-Stranded and Long, Partially Single-Stranded DNA Donors in Caenorhabditis elegans. Dokshin GA, Ghanta KS, Piscopo KM, and Mello CC. GENETICS. Early online September 13, 2018; doi.org/10.1534/genetics.118.301532
September 12, 2018, Anastasia Khvorova presents a webinar for the Oligonucleotide Therapeutics Society. Presentation now online: 'Oligonucleotide Therapeutics: Technology Evolution for Clinically Relevant Tissue Delivery'
September 11, 2018, New publication from the Grunwald lab. Fluorescence Polarization Control for On-Off Switching of Single Molecules at Cryogenic Temperatures. Hulleman CN, Huisman M, Moerland RJ, Grünwald D, Stallinga S, Rieger B.Small Methods. 2018 Sep 11;2(9). pii: 1700323. doi: 10.1002/smtd.201700323. Epub 2018 Apr 30. PMID:31240238
September 5, 2018 Congratulations to Enzhi Shen from the Mello Lab for being awarded a Charles A. King Trust Postdoctoral Fellowship in Basic Science!
August 14, 2018, Senator Markey visits UMass Chan Medical to speak with RTI researchers, Dr. Craig Mello, Dr. Anastasia Khvorova and Chancellor Michael Collins
August 13, 2018, New genome-editing strategy developed at UMass Chan may lead to therapeutics Dan Wang, Guangping Gao, Wen Xue, and Phillip D. Zamore describe this approach in Nature Biotechnology.
August 13, 2018, New publication from Xue, Zamore, and Gao labs. Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. Wang D, Li J, Song CQ, Tran K, Mou H, Wu PH, Tai PWL, Mendonca CA, Ren L, Wang BY, Su Q, Gessler DJ, Zamore PD, Xue W & Gao G. Nat Biotechnol. 13 Aug 2018. doi:10.1038/nbt.4219
August 10, 2018, FDA approves the first drug to use RNA interference, based on discoveries made at UMass Chan Medical School: The new drug, patisiran, approved on Aug. 10 by the FDA, is based on the discovery of RNAi made by Craig Mello, Ph.D., and Andrew Fire, Ph.D. It was developed by Alnylam, an RNAi-based drug development company co-founded by Phillip Zamore, Ph.D
August 10, 2018, New publication from Korostelev lab. Mechanism of premature translation termination on a sense codon Svidritskiy E, Demo G, Korostelev AA. J Biol Chem. 2018 Aug 10;293(32):12472-12479. doi: 10.1074/jbc.AW118.003232. Epub 2018 Jun 25.
August 7, 2018, New publication from the Khvorova lab. Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier. Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Mol Therapy August 07, 2018 DOI:https://doi.org/10.1016/j.ymthe.2018.08.005
August 5, 2018 Congratulations to Raed Ibraheim from the Sontheimer Lab for being awarded an NIH F31 Fellowship!
August 4, 2018 Congratulations, Zack Kennedy, from the Xue lab, upon receiving an Oligonucleotide Therapeutics Society travel grant!
August 1, 2018, New publication from Xue lab. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency Human Gene Therapy. Aug 2018. http://doi.org/10.1089/hum.2017.225
August 1, 2018, New publication from Zamore lab. Analysis of Small RNAs by Northern Hybridization Li C, Zamore PD. Cold Spring Harb Protoc. 2018 Aug 1;2018(8):pdb.prot097493. doi: 10.1101/pdb.prot097493.
July 31, 2018 Update from the Moore lab. Congratulations, Dr. Mihir Metkar Ph.D. for a successful thesis defense with the guidance of Drs. Moore and Dekker! 
July 13, 2018, New Publication from Zamore lab. Elimination of PCR duplicates in RNA-seq and small RNA-seq using unique molecular identifiers Yu Fu, Pei-Hsuan Wu, Timothy Beane, Phillip D. Zamore and Zhiping Weng. BMC Genomics. 2018 Jul 13;19(1):531. doi: 10.1186/s12864-018-4933-1.
July 6, 2018, New publication from Khvorova, Watts & Sontheimer labs. Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing Aamir Mir, Julia F. Alterman, Matthew R. Hassler, Alexandre J. Debacker, Edward Hudgens, Dimas Echeverria, Michael H. Brodsky, Anastasia Khvorova, Jonathan K. Watts & Erik J. Sontheimer. Nat Commun. 2018 Jul 6;9(1):2641. doi: 10.1038/s41467-018-05073-z.
July 5, 2018 Congratulations to Tomás Rodriguez from the Sontheimer Lab for being awarded Research Supplements to Promote Diversity in Health-Related Research from the NIDDK!
July 5, 2018 Congratulations to Ankur Sheel from the Xue Lab for being awarded an NIH F30 fellowship!
JUNE 14, 2018, New publication from the Khvorova lab. Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo. Sharma VK, Osborn MF, Hassler M, Echeverria D, Ly S, Ulashchik E, Martynenko-Makaev Y, Shmanai VV, Zatsepin TS, Khvorova A, Watts J.Bioconjug Chem. 2018 Jun 13. Bioconjugate Chem. 2018, 29, 7, 2478-2488
MAY 11, 2018, New publication from the Khvorova lab. Improving Small Interfering RNA Delivery In Vivo Through Lipid Conjugation. Osborn MF, and Khvorova A. doi.org/10.1089/nat.2018.0725
MAY 10, 2018, New publication from the Khvorova lab. Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs. Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, and Khvorova A. Molecular Cancer Therapeutics (2018) 10.1158/1535-7163.MCT-17-1144
MAY 7, 2018, New publication from the Sontheimer lab. C-BERST: defining subnuclear proteomic landscapes at genomic elements with dCas9–APEX2. Gao XD, Tu LC, Mir A, Rodriguez T, Ding Y, Leszyk J, Dekker J, Shaffer SA, Zhu LJ, Wolfe SA, & Sontheimer EJ. Nature Methods (2018) doi:10.1038/s41592-018-0006-2
May 4, 2018, Congratulations to Dr. Anastasia Khvorova and Dr. Andrei Korostelev for receiving tenure! This is a very fitting acknowledgment for both due to their hard work, fantastic mentorship, and scholarly contributions.
MAY 3, 2018, New publication from the Korostelev lab. Conformational Control of Translation Termination on The 70S Ribosome. Svidritskiy E, Korostelev AA. Structure. 2018 Apr 23. pii: S0969-2126(18)30126-6. doi: 10.1016/j.str.2018.04.001.
APRIL 23, 2018, News from the Khvorova lab. Congratulations, Dr. Reka Haraszti for successfully defending her thesis, the first to graduate from Dr. Anastasia Khvorova's lab!
APRIL 20, 2018, News from the Zamore lab. Congratulations, Dr. Timothy Chang, MD/Ph.D. student for successfully defending his thesis!
MARCH 29, 2018, New publication from the Xue lab. In vivo genome editing partially restores alpha1-antitrypsin in a murine model of AAT deficiency. Human Gene Therapy 29 March 2018; Dr. Chun-Qing Song, Dr. Dan Wang, Dr. Tingting Jiang, Mr. Kevin O'Connor, Dr. Qiushi Tang, Ms. Lingling Cai, Ms. Xiangrui Li, Dr. Zhiping Weng, Dr. Hao Yin, Dr. Guangping Gao, Dr. Christian Mueller, Dr. Terence R Flotte, Dr. Wen Xue. https://doi.org/10.1089/hum.2017.225
MARCH 28, 2018 Congratulations Xin Daniel Gao, Sontheimer lab for receiving the 2017 - 2018 Fall - Winter Riccio-Morningside Graduate School of Biomedical Sciences Travel Award!
MARCH 6, 2018, New editorial from the Khvorova lab. Nucleic Acids Research and Nucleic Acid Therapeutics. Nucleic Acids Res. 2018 Feb 28;46(4):1563-1564. Stoddard BL, Khvorova A, Corey DR, Dynan WS, Fox KR. doi: 10.1093/nar/gky059.
FEBRUARY 20, 2018, New publication from the Sontheimer lab. CRISPRs from scratch. Nature Microbiology 3, 261–262. Edraki, A, Sontheimer, E. doi:10.1038/s41564-018-0115-9
FEBRUARY 14, 2018, New publication from the Xue lab. CRISPR–Cas-related technologies in basic and translational liver research. Nature Reviews Gastroenterology & Hepatology. 2018. Song C-Q, Xue, W. doi:10.1038/nrgastro.2018.11
FEBRUARY 14, 2018, New publication from the Mello lab. The Coding Regions of Germline mRNAs Confer Sensitivity to Argonaute Regulation in C. elegans. Seth M, Shirayama M, Tang W, Shen EZ, Tu S, Lee HC, Weng Z, Mello CC. Cell Rep. 2018 Feb 14. pii: S2211-1247(18)30177-3. doi: 10.1016/j.celrep.2018.02.009.
FEBRUARY 13, 2018, New publication from the Mello lab. A Sex Chromosome piRNA Promotes Robust Dosage Compensation and Sex Determination in C. elegans. Tang W, Seth M, Tu S, Shen EZ, Li Q, Shirayama M, Weng Z, Mello CC. Dev Cell. 2018 Feb 13. pii: S1534-5807(18)30060-1. doi: 10.1016/j.devcel.2018.01.025.
FEBRUARY 10, 2018, New publication from the Mello lab. Identification of piRNA Binding Sites Reveals the Argonaute Regulatory Landscape of the C. elegans Germline. Shen EZ, Chen H, Ozturk AR, Tu S, Shirayama M, Tang W, Ding YH, Dai SY, Weng Z, Mello CC. Cell. 2018 Feb 10. pii: S0092-8674(18)30117-X. doi: 10.1016/j.cell.2018.02.002
FEBRUARY 8, 2018, New publication from the Khvorova lab. Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo. Hassler MR, Turanov AA, Alterman JF, Haraszti RA, Coles AH, Osborn MF, Echeverria D, Nikan M, Salomon WE, Roux L, Godinho BMDC, Davis SM, Morrissey DV, Zamore PD, Karumanchi SA, Moore MJ, Aronin N, Khvorova A. Nucleic Acids Research, gky037, https://doi.org/10.1093/nar/gky037
FEBRUARY 2, 2018, New publication from the Aronin & Khvorova lab. Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs. Didiot MC, Haraszti RA, Aronin N, Khvorova A.Methods Mol Biol. 2018;1740:199-214. doi: 10.1007/978-1-4939-7652-2_16
JANUARY 29, 2018, New publication from the Zamore lab. The genome of the Hi5 germ cell line from Trichoplusia ni, an agricultural pest and novel model for small RNA biology. Fu Y, Yang Y, Zhang H, Farley G, Wang J, Quarles KA, Weng Z, Zamore PD. Elife. 2018 Jan 29;7. pii: e31628. doi: 10.7554/eLife.31628. [Epub ahead of print] PMID: 29376823 [PubMed - as supplied by publisher]
JANUARY 26, 2018, New publication from the Korostelev lab. Mechanism of Inhibition of Translation Termination by Blasticidin S. Svidritskiy E, Korostelev AA. J Mol Biol. 2018 Jan 19. pii: S0022-2836(18)30010-X. doi: 10.1016/j.jmb.2018.01.007.
JANUARY 18, 2018, New publication from the Watts lab. Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat. Li L, Shen X, Liu Z, Norrbom M, Prakash TP, O'Reilly D, Sharma VK, Damha MJ, Watts JK, Rigo F, Corey DR. Nucleic Acid Ther. 2018 Jan 17. doi: 10.1089/nat.2017.0703.
JANUARY 8, 2018, New publication from the Watts lab. Oligonucleotides Targeting TCF4 Triplet Repeat Expansion Inhibit RNA Foci and Mis-splicing in Fuchs' Dystrophy. Hu J, Rong Z, Gong X, Zhou Z, Sharma VK, Xing C, Watts JK, Corey DR, Mootha VV. Hum Mol Genet. 2018 Jan 8. doi: 10.1093/hmg/ddy018.