RNA Therapeutics Institute Publications

 
The RNA Therapeutics Institute (RTI) at the University of Massachusetts Medical School was founded in 2009 and became an academic department in 2016, chaired by Phillip D. Zamore, Ph.D. The RTI is dedicated to leveraging the strong RNA biology and clinical research communities at UMMS to develop novel therapies for which RNA is the therapeutic target or modality. The RNA Therapeutics Institute faculty are recognized as scientific trailblazers, and include a Nobel Laureate, a Gairdner Prize recipient, the 2018 RNA Society early and mid-career award recipients, and two Howard Hughes Medical Institute Investigators. This collection showcases journal articles and other publications authored by RTI faculty and researchers.

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Publications from 2020

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Understanding the Production and Stability of Mouse PIWI-Interacting RNAs, Cansu Colpan

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Structure of the Vesicular Stomatitis Virus L Protein in Complex with Its Phosphoprotein Cofactor, Simon Jenni, Louis-Marie Bloyet, Ruben Diaz-Avalos, Bo Liang, Sean P.J. Whelan, Nikolaus Grigorieff, and Stephen C. Harrison

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Evolutionarily conserved pachytene piRNA loci are highly divergent among modern humans, Deniz M. Ozata, Tianxiong Yu, Haiwei Mou, Ildar Gainetdinov, Cansu Colpan, Katharine Cecchini, Yasin Kaymaz, Pei-Hsuan Wu, Kaili Fan, Alper Kucukural, Zhiping Weng, and Phillip D. Zamore

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Data on enrichment of chitosan nanoparticles for intranasal delivery of oligonucleotides to the brain, Vasyl Sava, Oksana Fihurka, Anastasia Khvorova, and Juan Sanchez-Ramos

Publications from 2019

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A CNS-Active siRNA Chemical Scaffold for the Treatment of Neurodegenerative Diseases, Julia F. Alterman

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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system, Julia F. Alterman, Bruno M. D. C. Godinho, Matthew R. Hassler, Chantal M. Ferguson, Dimas Echeverria, Ellen Sapp, Reka A. Haraszti, Andrew H. Coles, Faith Conroy, Rachael Miller, Loic Roux, Paul Yan, Emily G. Knox, Anton A. Turanov, Robert M. King, Gwladys Gernoux, Christian Mueller, Heather Gray-Edwards, Richard P. Moser, N Bishop, Samer M. Jaber, Matthew J. Gounis, Miguel Sena-Esteves, Athma A. Pai, Marian DiFiglia, Neil Aronin, and Anastasia Khvorova

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Ninjurin1 positively regulates osteoclast development by enhancing the survival of prefusion osteoclasts, Sung-Jin Bae, Min Wook Shin, Taekwon Son, Hye Shin. Lee, Ji Soo Chae, Sejin Jeon, Goo Taeg Oh, and Kyu-Won Kim

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Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo, Annabelle Biscans, Andrew H. Coles, Reka A. Haraszti, Dimas Echeverria, Matthew R. Hassler, Maire F. Osborn, and Anastasia Khvorova

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Compact Cas9s and Their Natural Inhibitors for Genome Editing, Alireza Edraki

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A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing, Alireza Edraki, Aamir Mir, Raed Ibraheim, Ildar Gainetdinov, Yeonsoo Yoon, Chun-Qing Song, Yueying Cao, Judith Gallant, Wen Xue, Jaime A. Rivera, and Erik J. Sontheimer

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Combining CRISPR-Cas9 and Proximity Labeling to Illuminate Chromatin Composition, Organization, and Regulation, Xin D. Gao

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Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing, Bianca Garcia, Jooyoung Lee, Alireza Edraki, Yurima Hidalgo-Reyes, Steven Erwood, Aamir Mir, Chantel N. Trost, Uri Seroussi, Sabrina Y. Stanley, Ronald D. Cohn, Julie M. Claycomb, Erik J. Sontheimer, Karen L. Maxwell, and Alan R. Davidson

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The RNA-Binding ATPase, Armitage, Couples piRNA Amplification in Nuage to Phased piRNA Production on Mitochondria, Daniel Tianfang Ge, Wei Wang, Cindy Tipping, Ildar Gainetdinov, Zhiping Weng, and Phillip D. Zamore

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Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition, Reka A. Haraszti, Rachael Miller, Michelle L. Dubuke, Andrew H. Coles, Marie C. Didiot, Dimas Echeverria, Matteo Stoppato, Yves Y. Sere, John D. Leszyk, Julia F. Alterman, Bruno M. D. C. Godinho, Matthew R. Hassler, Rachel Wollacott, Yan Wang, Scott A. Shaffer, Neil Aronin, and Anastasia Khvorova

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Vision Beyond Optics: Standardization, Evaluation and Innovation for Fluorescence Microscopy in Life Sciences, Maximiliaan Huisman

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In situ Structure of Rotavirus VP1 RNA-Dependent RNA Polymerase, Simon Jenni, Eric N. Salgado, Tobias Herrmann, Zongli Li, Timothy Grant, Nikolaus Grigorieff, Stefano Trapani, Leandro F. Estrozi, and Stephen C. Harrison

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A DNA-guided Argonaute Protein Functions in DNA Replication in Thermus thermophilus, Samson M. Jolly, Ildar Gainetdinov, , Han Zhang, Lara Strittmatter, Gregory M. Hendricks, Avantika Dhabaria, Beatrix Ueberheide, and Phillip D. Zamore

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High-resolution cryo-EM structures of outbreak strain human norovirus shells reveal size variations, James Jung, Timothy Grant, Dennis R. Thomas, Chris W. Diehnelt, Nikolaus Grigorieff, and Leemor Joshua-Tor

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Optimizing CRISPR/Cas9 for Gene Silencing of SOD1 in Mouse Models of ALS, Zachary C. Kennedy

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Deep sequencing of pre-translational mRNPs reveals hidden flux through evolutionarily conserved AS-NMD pathways, Carrie Kovalak, Mihir Metkar, and Melissa J. Moore

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DEBrowser: interactive differential expression analysis and visualization tool for count data, Alper Kucukural, Onur Yukselen, Deniz M. Ozata, Melissa J. Moore, and Manuel Garber

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Depletion of TRRAP induces p53-independent senescence in liver cancer by downregulating mitotic genes, Suet-Yan Kwan, Ankur Sheel, Chun-Qing Song, Xiao-Ou Zhang, Tingting Jiang, Hien Dang, Yueying Cao, Deniz M. Ozata, Haiwei Mou, Hao Yin, Zhiping Weng, Xin Wei Wang, and Wen Xue

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Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins, Jooyoung Lee, Haiwei Mou, Raed Ibraheim, Shun-Qing Liang, Pengpeng Liu, Wen Xue, and Erik J. Sontheimer

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CRISPR-SONIC: targeted somatic oncogene knock-in enables rapid in vivo cancer modeling, Haiwei Mou, Deniz M. Ozata, Jordan L. Smith, Ankur Sheel, Suet-Yan Kwan, Soren Hough, Alper Kucukural, Zachary Kennedy, Yueying Cao, and Wen Xue

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Improving the Health Benefits of Snap Bean: Genome-Wide Association Studies of Total Phenolic Content, James R. Myers, Lyle T. Wallace, Samira Mafi Moghaddam, Adrienne E. Kleintop, Dimas Echeverria, Henry J. Thompson, Mark A. Brick, Rian Lee, and Phillip E. McClean

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Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways, Maire F. Osborn, Andrew H. Coles, Annabelle Biscans, Reka A. Haraszti, Loic Roux, Sarah M. Davis, Socheata Ly, Dimas Echeverria, Matthew R. Hassler, Bruno M. D. C. Godinho, Mehran Nikan, and Anastasia Khvorova

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Intron and Small RNA Localization in Mammalian Neurons, Harleen Saini

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Free circular introns with an unusual branchpoint in neuronal projections, Harleen Saini, Alicia A. Bicknell, Sean R. Eddy, and Melissa J. Moore

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Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 In Vivo, Meirav Segal, Annabelle Biscans, Maud-Emmanuelle Gilles, Eleni Anastasiadou, Roberto De Luca, Jihoon Lim, Anastasia Khvorova, and Frank J. Slack

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Identification of Essential Genes in Hepatocellular Carcinomas using CRISPR Screening, Ankur Sheel

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An automated Bayesian pipeline for rapid analysis of single-molecule binding data, Carlas Smith, Karina Jouravleva, Maximiliaan Huisman, Samson M. Jolly, Phillip D. Zamore, and David Grünwald

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Principles for enhancing virus capsid capacity and stability from a thermophilic virus capsid structure, Nicholas P. Stone, Gabriel Demo, Emily Agnello, and Brian A. Kelch

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Principles for enhancing virus capsid capacity and stability from a thermophilic virus capsid structure, Nicholas P. Stone, Gabriel Demo, Emily Agnello, and Brian A. Kelch

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Extensive ribosome and RF2 rearrangements during translation termination, Egor Svidritskiy, Gabriel Demo, Anna B. Loveland, Chen Xu, and Andrei A. Korostelev

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Structure and assembly of calcium homeostasis modulator proteins, Johanna L. Syrjanen, Kevin Michalski, Tsung-Han Chou, Timothy Grant, Shanlin Rao, Noriko Simorowski, Stephen J. Tucker, Nikolaus Grigorieff, and Hiro Furukawa

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Inhibition of CRISPR-Cas9 ribonucleoprotein complex assembly by anti-CRISPR AcrIIC2, Annoj Thavalingam, Erik J. Sontheimer, Yanli Wang, and Karen L. Maxwell

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Gene Silencing With siRNA (RNA Interference): A New Therapeutic Option During Ex Vivo Machine Liver Perfusion Preservation, Max F. Thijssen, Isabel M. A. Bruggenwirth, Andrew R. Gillooly, Anastasia Khvorova, Timothy F. Kowalik, and Paulo N.A. Martins

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Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease, Adelaide Tousley, Anastasia Khvorova, Neil Aronin, and Kimberly B. Kegel-Gleason

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Targeted Metabolomics Identifies the Cytochrome P450 Monooxygenase Eicosanoid Pathway as a Novel Therapeutic Target of Colon Tumorigenesis, Weicang Wang, Jun Yang, Matthew L. Edin, Yuxin Wang, Ying Luo, Debin Wan, Haixia Yang, Chun-Qing Song, Wen Xue, Katherine Z. Sanidad, Mingyue Song, Heather A. Bisbee, Jennifer A. Bradbury, Guanjun Nan, Jianan Zhang, Pei-An Betty Shih, Kin Sing Stephen Lee, Lisa M. Minter, Daeyoung Kim, Hang Xiao, Jun-Yan Liu, Bruce D. Hammock, Darryl C. Zeldin, and Guodong Zhang

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IIV-6 Inhibits NF-kappaB Responses in Drosophila, Cara C. West, Florentina Rus, Ying Chen, Anni Kleino, Monique Gangloff, Don B. Gammon, and Neal S. Silverman

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HSP90/AXL/eIF4E-regulated unfolded protein response as an acquired vulnerability in drug-resistant KRAS-mutant lung cancer, Haitang Yang, Shun-Qing Liang, Duo Xu, Zhang Yang, Thomas M. Marti, Yanyun Gao, Gregor J. Kocher, Heng Zhao, Ralph A. Schmid, and Ren-Wang Peng

Publications from 2018

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Bayesian nonparametric discovery of isoforms and individual specific quantification, Derek Aguiar, Li-Fang Cheng, Bianca Dumitrascu, Fantine Mordelet, Athma A. Pai, and Barbara E. Engelhardt

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NmeCas9 is an intrinsically high-fidelity genome editing platform, Nadia Amrani, Xin D. Gao, Pengpeng Liu, Alireza Edraki, Aamir Mir, Raed Ibraheim, Ankit Gupta, Kanae E. Sasaki, Tong Wu, Thomas G. Fazzio, Lihua Julie Zhu, Scot A. Wolfe, and Erik J. Sontheimer

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NmeCas9 is an intrinsically high-fidelity genome-editing platform, Nadia Amrani, Xin D. Gao, Pengpeng Liu, Alireza Edraki, Aamir Mir, Raed Ibraheim, Ankit Gupta, Kanae E. Sasaki, Tong Wu, Thomas G. Fazzio, Lihua Julie Zhu, Scot A. Wolfe, and Erik J. Sontheimer

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Exploring the Emergence of RNA Nucleosides and Nucleotides on the Early Earth, Annabelle Biscans

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Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles, Annabelle Biscans, Reka A. Haraszti, Dimas Echeverria, Rachael Miller, Marie C. Didiot, Mehran Nikan, Loic Roux, Neil Aronin, and Anastasia Khvorova

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Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing, Mehmet F. Bolukbasi, Pengpeng Liu, Kevin Luk, Samantha F. Kwok, Ankit Gupta, Nadia Amrani, Erik J. Sontheimer, Lihua Julie Zhu, and Scot A. Wolfe

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Synergistic assembly of human pre-spliceosomes across introns and exons, Joerg E. Braun, Larry J. Friedman, Jeff Gelles, and Melissa J. Moore

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Maelstrom Represses Canonical RNA Polymerase II Transcription in Drosophila Dual-Strand piRNA Clusters, Timothy H. Chang

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Maelstrom Represses Canonical Polymerase II Transcription within Bi-directional piRNA Clusters in Drosophila melanogaster, Timothy Chang, Eugenio Mattei, Ildar Gainetdinov, Cansu Colpan, Zhiping Weng, and Phillip D. Zamore

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Transcriptome-wide Interrogation of the Functional Intronome by Spliceosome Profiling, Weijun Chen, Jill E. Moore, Hakan Ozadam, Hennady P. Shulha, Nicholas Rhind, Zhiping Weng, and Melissa J. Moore

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Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin, Marie C. Didiot, Chantal M. Ferguson, Socheata Ly, Andrew H. Coles, Abigail O. Smith, Alicia A. Bicknell, Lauren M. Hall, Ellen Sapp, Dimas Echeverria, Athma A. Pai, Marian DiFiglia, Melissa J. Moore, Lawrence J. Hayward, Neil Aronin, and Anastasia Khvorova

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Robust Genome Editing with Short Single-Stranded and Long, Partially Single-Stranded DNA Donors in Caenorhabditis elegans, Gregoriy A. Dokshin, Krishna S. Ghanta, Katherine M. Piscopo, and Craig C. Mello

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Elimination of PCR duplicates in RNA-seq and small RNA-seq using unique molecular identifiers, Yu Fu, Pei-Hsuan Wu, Timothy J. Beane, Phillip D. Zamore, and Zhiping Weng

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Elimination of PCR duplicates in RNA-seq and small RNA-seq using unique molecular identifiers, Yu Fu, Pei-Hsuan Wu, Timothy J. Beane, Phillip D. Zamore, and Zhiping Weng

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The genome of the Hi5 germ cell line from Trichoplusia ni, an agricultural pest and novel model for small RNA biology, Yu Fu, Yujing Yang, Han Zhang, Gwen Farley, Junling Wang, Kaycee A. Quarles, Zhiping Weng, and Phillip D. Zamore

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C-BERST: defining subnuclear proteomic landscapes at genomic elements with dCas9-APEX2, Xin D. Gao, Li-Chun Tu, Aamir Mir, Tomas Rodriguez, Yue-He Ding, John D. Leszyk, Job Dekker, Scott A. Shaffer, Lihua Julie Zhu, Scot A. Wolfe, and Erik J. Sontheimer (Nature methods, 2018-05-07)

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C-BERST: Defining subnuclear proteomic landscapes at genomic elements with dCas9-APEX2, Xin D. Gao, Li-Chun Tu, Aamir Mir, Tomas Rodriguez, Yue-He Ding, John D. Leszyk, Job Dekker, Scott A. Shaffer, Lihua Julie Zhu, Scot A. Wolfe, and Erik J. Sontheimer

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Understanding Nuage-mitochondrial Coupling in Drosophila piRNA Biogenesis, Tianfang Ge

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Engineered Exosomes for Delivery of Therapeutic siRNAs to Neurons, Reka A. Haraszti

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Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles, Reka A. Haraszti, Rachael Miller, Marie C. Didiot, Annabelle Biscans, Julia F. Alterman, Matthew R. Hassler, Loic Roux, Dimas Echeverria, Ellen Sapp, Marian DiFiglia, Neil Aronin, and Anastasia Khvorova

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Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity, Reka A. Haraszti, Rachael Miller, Matteo Stoppato, Yves Y. Sere, Andrew H. Coles, Marie-Claire Daou, Rachel Wollacott, Ellen Sapp, Michelle L. Dubuke, Xuni Li, Scott A. Shaffer, Marian DiFiglia, Yan Wang, Neil Aronin, and Anastasia Khvorova

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Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo, Matthew R. Hassler, Anton A. Turanov, Julia F. Alterman, Reka A. Haraszti, Andrew H. Coles, Maire F. Osborn, Dimas Echeverria, Mehran Nikan, William E. Salomon, Loic Roux, Bruno M. D. C. Godinho, Sarah M. Davis, David V. Morrissey, Phillip D. Zamore, S. Ananth Karumanchi, Melissa J. Moore, Neil Aronin, and Anastasia Khvorova

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Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs' dystrophy, Jiaxin Hu, Ziye Rong, Xin Gong, Zhengyang Zhou, Vivek K. Sharma, Chao Xing, Jonathan K. Watts, David R. Corey, and V. Vinod Mootha

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Fluorescence Polarization Control for On-Off Switching of Single Molecules at Cryogenic Temperatures, Christiaan N. Hulleman, Maximiliaan Huisman, Robert J. Moerland, David Grünwald, Sjoerd Stallinga, and Bernd Rieger

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All-in-One Adeno-associated Virus Delivery and Genome Editing by Neisseria meningitidis Cas9 in vivo, Raed Ibraheim, Chun-Qing Song, Aamir Mir, Nadia Amrani, Wen Xue, and Erik J. Sontheimer

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All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo, Raed Ibraheim, Chun-Qing Song, Aamir Mir, Nadia Amrani, Wen Xue, and Erik J. Sontheimer

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ZNFX-1 Functions within Perinuclear Nuage to Balance Epigenetic Signals, Takao Ishidate, Ahmet R. Ozturk, Daniel J. Durning, Rita Sharma, En-Zhi Shen, Hao Chen, Meetu Seth, Masaki Shirayama, and Craig C. Mello

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PIE-1, SUMOylation, and Epigenetic Regulation of Germline Specification in Caenorhabditis elegans, Heesun Kim

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Potent Cas9 Inhibition in Bacterial and Human Cells by AcrIIC4 and AcrIIC5 Anti-CRISPR Proteins, Jooyoung Lee, Aamir Mir, Alireza Edraki, Bianca Garcia, Nadia Amrani, Hannah E. Lou, Ildar Gainetdinov, April Pawluk, Raed Ibraheim, Xin D. Gao, Pengpeng Liu, Alan R. Davidson, Karen L. Maxwell, and Erik J. Sontheimer

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Pan-arthropod analysis reveals somatic piRNAs as an ancestral defence against transposable elements, Samuel H. Lewis, Yujing Yang, Phillip D. Zamore, and Francis M. Jiggins

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Structural dynamics of protein S1 on the 70S ribosome visualized by ensemble cryo-EM, Anna B. Loveland and Andrei A. Korostelev

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Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs, Yi Lu, Phillip W. L. Tai, Jianzhong Ai, Dominic J. Gessler, Qin Su, Xieyi Yao, Qiang Zheng, Phillip D. Zamore, Xun Xu, and Guangping Gao

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CRISPR-Sirius: RNA scaffolds for signal amplification in genome imaging, Hanhui Ma, Li-Chun Tu, Ardalan Naseri, Yu-Chieh Chung, David Grünwald, Shaojie Zhang, and Thoru Pederson

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RIPPLiT and ChimeraTie: High throughput tools for understanding higher order RNP structures, Mihir Metkar

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Higher-Order Organization Principles of Pre-translational mRNPs, Mihir Metkar, Hakan Ozadam, Bryan R. Lajoie, Maxim Imakaev, Leonid A. Mirny, Job Dekker, and Melissa J. Moore

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Higher-Order Organization Principles of Pre-translational mRNPs, Mihir Metkar, Hakan Ozadam, Bryan R. Lajoie, Maxim Imakaev, Leonid A. Mirny, Job Dekker, and Melissa J. Moore (Molecular cell, 2018-11-15)

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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, and Erik J. Sontheimer

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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, and Erik J. Sontheimer

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Type II-C CRISPR-Cas9 Biology, Mechanism, and Application, Aamir Mir, Alireza Edraki, Jooyoung Lee, and Erik J. Sontheimer

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Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs, Maire F. Osborn, Andrew H. Coles, Diane Golebiowski, Dimas Echeverria, Michael P. Moazami, Jonathan K. Watts, Miguel Sena-Esteves, and Anastasia Khvorova

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Pof8 is a La-related protein and a constitutive component of telomerase in fission yeast, Diego J. Paez-Moscoso, Lili Pan, Rutendo F. Sigauke, Morgan R. Schroeder, Wen Tang, and Peter Baumann

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Numerous recursive sites contribute to accuracy of splicing of long introns in flies, Athma A. Pai, Joseph Paggi, Karen Adelman, and Christopher B. Burge

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Numerous recursive sites contribute to accuracy of splicing in long introns in flies, Athma A. Pai, Joseph M. Paggi, Paul Yan, Karen Adelman, and Christopher B. Burge

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A Fresh Look at Huntingtin mRNA Processing in Huntington's Disease, Lindsay S. Romo, Emily S. Mohn, and Neil Aronin

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Extracellular vesicles from mice with alcoholic liver disease carry a distinct protein cargo and induce macrophage activation through heat shock protein 90, Banishree Saha, Fatemeh Momen-Heravi, Istvan Furi, Karen Kodys, Donna Catalano, Anwesha Gangopadhyay, Reka A. Haraszti, Abhishek Satishchandran, Arvin Iracheta-Vellve, Adeyinka Adejumo, Scott A. Shaffer, and Gyongyi Szabo

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Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain, Juan Sanchez-Ramos, Shijie Song, Xiaoyuan Kong, Parastou Foroutan, Gary Martinez, William Dominguez-Viqueria, Shyam Mohapatra, Subhra Mohapatra, Reka A. Haraszti, Anastasia Khvorova, Neil Aronin, and Vasyl Sava

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The Coding Regions of Germline mRNAs Confer Sensitivity to Argonaute Regulation in C. elegans, Meetu Seth, Masaki Shirayama, Wen Tang, En-Zhi Shen, Shikui Tu, Heng-Chi Lee, Zhiping Weng, and Craig C. Mello

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Identification of piRNA Binding Sites Reveals the Argonaute Regulatory Landscape of the C. elegans Germline, En-Zhi Shen, Hao Chen, Ahmet R. Ozturk, Shikui Tu, Masaki Shirayama, Wen Tang, Yue-He Ding, Si-Yuan Dai, Zhiping Weng, and Craig C. Mello

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Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs, Taisia Shmushkovich, Kathryn R. Monopoli, Diana Homsy, Dmitriy Leyfer, Monica Betancur-Boissel, Anastasia Khvorova, and Alexey D. Wolfson

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An Automated Bayesian Pipeline for Rapid Analysis of Single-Molecule Binding Data, Carlas Smith, Karina Jouravleva, Maximiliaan Huisman, Samson M. Jolly, Phillip D. Zamore, and David Grünwald

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Understanding and repurposing CRISPR-mediated alternative splicing, Jordan L. Smith, Haiwei Mou, and Wen Xue

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In vivo genome editing partially restores alpha1-antitrypsin in a murine model of AAT deficiency, Chun-Qing Song, Dan Wang, Tingting Jiang, Kevin O'Connor, Qiushi Tang, Lingling Cai, Xiangrui Li, Zhiping Weng, Hao Yin, Guangping Gao, Christian Mueller, Terence R. Flotte, and Wen Xue

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CRISPR-Cas-related technologies in basic and translational liver research, Chun-Qing Song and Wen Xue

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Editorial: Nucleic Acids Research and Nucleic Acid Therapeutics, Barry L. Stoddard, Anastasia Khvorova, David R. Corey, William S. Dynan, and Keith R. Fox

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A Hyperthermophilic Phage Decoration Protein Suggests Common Evolutionary Origin with Herpesvirus Triplex Proteins and an Anti-CRISPR Protein, Nicholas P. Stone, Brendan J. Hilbert, Daniel Hidalgo, Kevin T. Halloran, Jooyoung Lee, Erik J. Sontheimer, and Brian A. Kelch

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Mechanism of premature translation termination on a sense codon, Egor Svidritskiy, Garbriel Demo, and Andrei A. Korostelev

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Mechanism of Inhibition of Translation Termination by Blasticidin S, Egor Svidritskiy and Andrei A. Korostelev

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A Sex Chromosome piRNA Promotes Robust Dosage Compensation and Sex Determination in C. elegans, Wen Tang, Meetu Seth, Shikui Tu, En-Zhi Shen, Qian Li, Masaki Shirayama, Zhiping Weng, and Craig C. Mello

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Minimal information for studies of extracellular vesicles 2018 (MISEV2018): a position statement of the International Society for Extracellular Vesicles and update of the MISEV2014 guidelines, Clotilde Thery, Kenneth W. Witwer, Reka A. Haraszti, and Anastasia Khvorova