Publications
Published research driving the development of next-generation therapeutic RNAi and ASO technologies
Publications
Published research driving the development of next-generation siRNA delivery technologies for therapeutic applications
Filters:
| Title | Author | Link | hf:file_type | hf:doc_categories | hf:doc_author | hf:doc_tags |
|---|---|---|---|---|---|---|
| Self-Delivering RNAi Compounds | Dmitry Samarsky, James Cardia, Tod Woolf | www | dmitry-samarsky james-cardia tod-woolf | |||
| Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs | Anastasia Khvorova, Andrew H Coles, Hong Cao, Julia F. Alterman, Maire F Osborn, Marie-Cécile Didiot, Matthew R. Hassler, Mehran Nikan | www | anastasia-khvorova andrew-h-coles hong-cao julia-f-alterman maire-f-osborn marie-cecile-didiot matthew-r-hassler mehran-nikan | |||
| A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo | Anastasia Khvorova, Andrew H Coles, Anton A Turanov, Bruno M. D. C. Godinho, Julia F. Alterman, Kathryn Chase, Lori Kennington, Maire F Osborn, Neil Aronin | www | anastasia-khvorova andrew-h-coles anton-a-turanov bruno-m-d-c-godinho julia-f-alterman kathryn-chase lori-kennington maire-f-osborn neil-aronin | |||
| Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye | Amanda Rodgers, Anastasia Khvorova, Glenna Ford, James Cardia, Jennifer Lapierre, Karen Bulock, Katherine Holton, Kevin Fettes, Lakshmipathi Pandarinathan, Lyn Libertine, Michael Byrne, Pamela Pavco, Radouil Tzekov, Shalesh Kaushal, Sharon Shaw, William Stanney, Yi Wang | www | amanda-rodgers anastasia-khvorova glenna-ford james-cardia jennifer-lapierre karen-bulock katherine-holton kevin-fettes lakshmipathi-pandarinathan lyn-libertine michael-byrne pamela-pavco radouil-tzekov shalesh-kaushal sharon-shaw william-stanney yi-wang | |||
| Exosomes are natural carriers of exogenous siRNA to human cells in vitro | Elena Yu Varfolomeeva, Evgeny M Makarov, Michael V Filatov, Roman A Kovalev, Tatyana A Shtam, Yury V Kil | www | elena-yu-varfolomeeva evgeny-m-makarov michael-v-filatov roman-a-kovalev tatyana-a-shtam yury-v-kil | |||
| Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain | Anastasia Khvorova, Andrew H Coles, Ellen Sapp, Emily Johnson, Emily Sottosanti, Jasmin Abraham, Julia F. Alterman, Kathryn Chase, Lauren M Hall, Maire F Osborn, Marian DiFiglia, Marie-Cécile Didiot, Matthew R. Hassler, Neil Aronin | www | anastasia-khvorova andrew-h-coles ellen-sapp emily-johnson emily-sottosanti jasmin-abraham julia-f-alterman kathryn-chase lauren-m-hall maire-f-osborn marian-difiglia marie-cecile-didiot matthew-r-hassler neil-aronin | |||
| Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain | Anastasia Khvorova, Andrew H Coles, Bruno M. D. C. Godinho, Dimas Echeverria, Lauren M Hall, Maire F Osborn, Matthew R. Hassler, Mehran Nikan, Neil Aronin, Reka A Haraszti | www | anastasia-khvorova andrew-h-coles bruno-m-d-c-godinho dimas-echeverria lauren-m-hall maire-f-osborn matthew-r-hassler mehran-nikan neil-aronin reka-a-haraszti | |||
| Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing | Anastasia Khvorova, Andrew H Coles, Bruno M. D. C. Godinho, David V Morrissey, Dimas Echeverria, Ellen Sapp, Julia F. Alterman, Kathryn Chase, Lakshmi Raj, Lauren M Hall, Marian DiFiglia, Marie-Cécile Didiot, Matthew R. Hassler, Neil Aronin, Reka A Haraszti, Socheata Ly | www | anastasia-khvorova andrew-h-coles bruno-m-d-c-godinho david-v-morrissey dimas-echeverria ellen-sapp julia-f-alterman kathryn-chase lakshmi-raj lauren-m-hall marian-difiglia marie-cecile-didiot matthew-r-hassler neil-aronin reka-a-haraszti socheata-ly | |||
| Visualization of self-delivering hydrophobically modified siRNA cellular internalization | Anastasia Khvorova, Clive Standley, Deanna M Navaroli, Dimas Echeverria, Julia F. Alterman, Karl D Bellve, Kevin Fogarty, Lakshmipathi Pandarinathan, Lawrence M Lifshitz, Marie-Cécile Didiot, Matthieu Prot, Socheata Ly | www | anastasia-khvorova clive-standley deanna-m-navaroli dimas-echeverria julia-f-alterman karl-d-bellve kevin-fogarty lakshmipathi-pandarinathan lawrence-m-lifshitz marie-cecile-didiot matthieu-prot socheata-ly | |||
| Oligonucleotide Therapeutics — A New Class of Cholesterol-Lowering Drugs | Anastasia Khvorova | www | anastasia-khvorova | |||
| The chemical evolution of oligonucleotide therapies of clinical utility | Anastasia Khvorova, Jonathan K Watts | www | anastasia-khvorova jonathan-k-watts | |||
| Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain | Anastasia Khvorova, Andrew H Coles, Annabelle Biscans, Bruno M. D. C. Godinho, Dimas Echeverria, Ellen Sapp, Maire F Osborn, Marian DiFiglia, Mehran Nikan, Neil Aronin, Reka A Haraszti | www | anastasia-khvorova andrew-h-coles annabelle-biscans bruno-m-d-c-godinho dimas-echeverria ellen-sapp maire-f-osborn marian-difiglia mehran-nikan neil-aronin reka-a-haraszti | |||
| Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System | Anastasia Khvorova, Andrew H Coles, Marie-Cécile Didiot, Neil Aronin, Reka A Haraszti | www | anastasia-khvorova andrew-h-coles marie-cecile-didiot neil-aronin reka-a-haraszti | |||
| A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics | Anastasia Khvorova, Andrew H Coles, Julia F. Alterman, Lauren M Hall, Marie-Cécile Didiot, Neil Aronin | www | anastasia-khvorova andrew-h-coles julia-f-alterman lauren-m-hall marie-cecile-didiot neil-aronin | |||
| Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay | Anastasia Khvorova, Andrew H Coles, Annabelle Biscans, Bruno M. D. C. Godinho, Dimas Echeverria, James W Gilbert, Loic Roux, Matthew R. Hassler, Mehran Nikan, Reka A Haraszti | www | anastasia-khvorova andrew-h-coles annabelle-biscans bruno-m-d-c-godinho dimas-echeverria james-w-gilbert loic-roux matthew-r-hassler mehran-nikan reka-a-haraszti | |||
| Manipulation of Panx1 Activity Increases the Engraftment of Transplanted Lacrimal Gland Epithelial Progenitor Cells | Alexey Wolfson, Anastasia Gromova, Dillon Hawley, Driss Zoukhri, Helen P Makarenkova, Liana V Basova, Taisia Shmushkovich, Takeshi Umasume, Tatiana Zyrianova, Valery I Shestopalov, Xin Tang | www | alexey-wolfson anastasia-gromova dillon-hawley driss-zoukhri helen-p-makarenkova liana-v-basova taisia-shmushkovich takeshi-umasume tatiana-zyrianova valery-i-shestopalov xin-tang | |||
| Self-Delivering RNAi Targeting PD-1 Improves Tumor-Specific T Cell Functionality for Adoptive Cell Therapy of Malignant Melanoma | Alexey D Wolfson, Alexey V Eliseev, Iva Truxova, Maarten A Ligtenberg, Monica Betancur-Boissel, Rolf Kiessling, Taisia Shmushkovich, Yago Pico de Coaña, Yuan Yang, Yuya Yoshimoto | www | alexey-d-wolfson alexey-v-eliseev iva-truxova maarten-a-ligtenberg monica-betancur-boissel rolf-kiessling taisia-shmushkovich yago-pico-de-coana yuan-yang yuya-yoshimoto | |||
| Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs | Alexey D Wolfson, Anastasia Khvorova, Diana Homsy, Dmitriy Leyfer, Kathryn R Monopoli, Monica Betancur-Boissel, Taisia Shmushkovich | www | alexey-d-wolfson anastasia-khvorova diana-homsy dmitriy-leyfer kathryn-r-monopoli monica-betancur-boissel taisia-shmushkovich | |||
| Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin | Abigail O Smith, Alicia A Bicknell, Andrew H Coles, Chantal M. Ferguson, Ellen Sapp, Marie-Cécile Didiot, Socheata Ly | www | abigail-o-smith alicia-a-bicknell andrew-h-coles chantal-m-ferguson ellen-sapp marie-cecile-didiot socheata-ly | |||
| Improving siRNA Delivery In Vivo Through Lipid Conjugation | Anastasia Khvorova, Maire F Osborn | www | anastasia-khvorova maire-f-osborn | |||
| A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system | Bruno M. D. C. Godinho, Chantal M. Ferguson, Dimas Echeverria, Ellen Sapp, Julia F. Alterman, Matthew R. Hassler | www | bruno-m-d-c-godinho chantal-m-ferguson dimas-echeverria ellen-sapp julia-f-alterman matthew-r-hassler | |||
| USP10 Targeted Self-Deliverable siRNA to Prevent Scarring in the Cornea | Andrew T. Phillips, Audrey M. Bernstein, Edward F. Boumil, Marc Ridilla, Nileyma Castro, Taisia Shmushkovich | www | andrew-t-phillips audrey-m-bernstein edward-f-boumil marc-ridilla nileyma-castro taisia-shmushkovich | |||
| Self-delivering RNAi compounds as therapeutic agents in the central nervous system to enhance axonal regeneration after injury | Alyson E. Fournier, Barbara Morquette, Joerg Ruschel, Kenneth M. Rosen, Lisa McKerracher, Sarah A. Woller | www | alyson-e-fournier barbara-morquette joerg-ruschel kenneth-m-rosen lisa-mckerracher sarah-a-woller | |||
| Prospects for RNAi Therapy of COVID-19 | Azita Haddadi, Hamidreza Montazeri Aliabadi, Hasan Uludağ, Kylie Parent | www | azita-haddadi hamidreza-montazeri-aliabadi hasan-uludag kylie-parent | |||
| siRNA could be a potential therapy for COVID-19 | Anirban Nath, Sanhita Ghosh, Sayeed Mohammad Firdous | www | anirban-nath sanhita-ghosh sayeed-mohammad-firdous |