Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT

Authors: Iwamoto, Naoki, Liu, Yuanjing, Frank-Kamenetsky, Maria, Maguire, Abbie, Tseng, Wei Chou, Taborn, Kristin, Kothari, Nayantara, Akhtar, Ali, Bowman, Keith, Shelke, Juili Dilip, Lamattina, Anthony, Hu, Xiao Shelley, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Ken, Looby, Richard, Meena, Metterville, Jake, Pan, Qianli, Purcell-Estabrook, Erin, Shimizu, Mamoru, Prakasha, Priyanka Shiva, Standley, Stephany, Upadhyay, Hansini, Yang, Hailin, Yin, Yuan, Zhao, Anderson, Francis, Christopher, Byrne, Mike, Dale, Elena, Verdine, Gregory L., Vargeese, Chandra

Source: In Molecular Therapy - Nucleic Acids 10 September 2024 35(3)

Quantitative Measurement of Cytosolic and Nuclear Penetration of Oligonucleotide Therapeutics

Authors: Deprey, Kirsten, Batistatou, Nefeli, Debets, Marjoke F., Godfrey, Jack, VanderWall, Kirstin B., Miles, Rebecca R., Shehaj, Livia, Guo, Jiaxing, Andreucci, Amy, Kandasamy, Pachamuthu

Source: ACS chemical biology. 17(2):348-360

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN628751510&indx=1&recIds=ETOCRN628751510

WVE‐005, a stereopure antisense oligonucleotide for MAPT silencing in tauopathies

Authors: DaleMaguire, ElenaAbbie, Guo, Lankai, Byrne, Michael, Cannon, Megan, Taborn, Kris, Prakasha, Priyanka Shiva, Longo, Kenneth, Iwamoto, Naoki, Kandasamy, Pachamuthu, Shimizu, Mamoru, Shum, Pochi, Ranade, Ashwini, Iyer, Raghuvaran, Bowman, Keith, Yang, Hailin, Yin, Yuan Benny, Liu, Fangjun, Mohapatra, Susovan, Macovei, Lilia, Patil, Saurabh, Lansita, Janice, Hu, Xiao Shelley, Smith, Karen, Goyal, Jaya, Narayanan, Padma, Rhodes, Ken, Panzara, Michael, Vargeese, Chandra

Source: Alzheimer's & dementia. 18(10)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100177004605.0x000001&indx=1&recIds=ETOCvdc_100177004605.0x000001

SiRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

Authors: Matsuda, Shigeo, Keiser, Kristofer, Nair, Jayaprakash K., Charisse, Klaus, Manoharan, Rajar M., Kretschmer, Philip, Peng, Chang G., Kel in, Alexander V., Kandasamy, Pachamuthu, Willoughby, Jennifer L. S.

Source: ACS chemical biology. 10(5):1181-1187

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN601251411&indx=1&recIds=ETOCRN601251411

FRI-151 RNA editing for the treatment of Alpha-1 antitrypsin deficiency

Authors: Monian, Prashant, Lu, Genliang, Shivalila, Chikdu, Bowman, Keith, Bylsma, Marissa, Byrne, Michael, Cannon, Megan, Desai, Jigar, Faraone, Alyse, Favaloro, Frank, Ghosh, Anamitra, Godfrey, Jack, Hernandez, Nidia, Huth, Olivia, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Kandasamy, Pachamuthu, Lamattina, Anthony, Lindsey, Amber, Liu, Fangjun, Looby, Richard, Luu, Khoa, Metterville, Jake, Paik, Ik-Hyeon, Pan, Qianli, Purcell-Estabrook, Erin, Rheinhardt, Jeanette, Shimizu, Mamoru, Singh, Kuldeep, Standley, Stephany, Thomas, Carina, Tripathi, Snehlata, Yang, Hailin, Yordanoff, Ryan, Yin, Benny, Yu, Hui, Caracta, Cynthia, Narayanan, Padma, Giangrande, Paloma, Vargeese, Chandra

Source: In Journal of Hepatology June 2024 80 Supplement 1:S692-S693

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

Authors: Maier, Martin A, Jayaraman, Muthusamy, Matsuda, Shigeo, Liu, Ju, Barros, Scott, Querbes, William, Tam, Ying K, Ansell, Steven M, Kumar, Varun, Qin, June, Zhang, Xuemei, Wang, Qianfan, Panesar, Sue, Hutabarat, Renta, Carioto, Mary, Hettinger, Julia, Kandasamy, Pachamuthu, Butler, David, Rajeev, Kallanthottathil G, Pang, Bo, Charisse, Klaus, Fitzgerald, Kevin, Mui, Barbara L, Du, Xinyao, Cullis, Pieter, Madden, Thomas D, Hope, Michael J, Manoharan, Muthiah, Akinc, Akin

Source: Molecular therapy. 21(8):1570-1578

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100056954230.0x000001&indx=1&recIds=ETOCvdc_100056954230.0x000001

Metabolically Stable Anomeric Linkages Containing GalNAc–siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways

Authors: Kandasamy, Pachamuthu, Mori, Shohei, Matsuda, Shigeo, Erande, Namrata, Datta, Dhrubajyoti, Willoughby, Jennifer L. S., Taneja, Nate, O’Shea, Jonathan, Bisbe, Anna, Manoharan, Rajar M., Yucius, Kristina, Nguyen, Tuyen, Indrakanti, Ramesh, Gupta, Swati, Gilbert, Jason A., Racie, Tim, Chan, Amy, Liu, Ju, Hutabarat, Renta, Nair, Jayaprakash K., Charisse, Klaus, Maier, Martin A., Rajeev, Kallanthottathil G., Egli, Martin, Manoharan, Muthiah

Source: Journal of Medicinal Chemistry; February 2023, Vol. 66 Issue: 4 p2506-2523, 18p

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity.

Authors: Kandasamy, Pachamuthu, McClorey, Graham, Shimizu, Mamoru, Kothari, Nayantara, Alam, Rowshon, Naoki Iwamoto, Kumarasamy, Jayakanthan, Bommineni, Gopal R., Bezigian, Adam, Chivatakarn, Onanong, Butler, David C. D., Byrne, Michael, Chwalenia, Katarzyna, Davies, Kay E., Desai, Jigar, Shelke, Juili Dilip, Durbin, Ann F., Ellerington, Ruth, Edwards, Ben, Godfrey, Jack

Source: Nucleic Acids Research; 6/10/2022, Vol. 50 Issue 10, p5443-5466, 24p, 1 Diagram, 10 Graphs

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.

Authors: Liu, Yuanjing, Dodart, Jean-Cosme, Tran, Helene, Berkovitch, Shaunna, Braun, Maurine, Byrne, Michael, Durbin, Ann F., Hu, Xiao Shelley, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Kenneth, Ruschel, Jörg, Shelke, Juili, Yang, Hailin, Yin, Yuan, Donner, Amy, Zhong, Zhong, Vargeese, Chandra

Source: Nature Communications; 2/8/2021, Vol. 12 Issue 1, p1-15, 15p

Subject Terms: ANIMAL models in research, AMYOTROPHIC lateral sclerosis, OLIGONUCLEOTIDES, MOTOR neuron diseases, FRONTOTEMPORAL dementia, MOTOR neurons, TRANSGENIC mice, FACIOSCAPULOHUMERAL muscular dystrophy

THE INVESTIGATIONAL STEREOPURE ANTISENSE OLIGONUCLEOTIDE WVE-3972-01 PREFERENTIALLY REDUCES G4C2 REPEAT-CONTAINING C9ORF72 TRANSCRIPTS: A POTENTIAL THERAPEUTIC APPROACH FOR THE TREATMENT OF FRONTOTEMPORAL DEMENTIA

Authors: Dodart, Jean-Cosme, Liu, Yuanjing, Tran, Helene, Berkovitch, Shaunna, Byrne, Michael, Durbin, Ann, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Mohapatra, Susovan, Yang, Hailin, Yin, Yuan, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.

Source: Alzheimer's & dementia. 14(7):P1411-P1411

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100069586241.0x000001&indx=1&recIds=ETOCvdc_100069586241.0x000001

O4‐05‐02: THE INVESTIGATIONAL STEREOPURE ANTISENSE OLIGONUCLEOTIDE WVE‐3972‐01 PREFERENTIALLY REDUCES G4C2 REPEAT‐CONTAINING C9ORF72 TRANSCRIPTS: A POTENTIAL THERAPEUTIC APPROACH FOR THE TREATMENT OF FRONTOTEMPORAL DEMENTIA

Authors: Dodart, Jean-Cosme, Liu, Yuanjing, Tran, Helene, Berkovitch, Shaunna, Byrne, Michael, Durbin, Ann, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Mohapatra, Susovan, Yang, Hailin, Yin, Yuan, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.

Source: Alzheimer's & dementia. 14(7):P1411-P1411

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100174732606.0x000001&indx=1&recIds=ETOCvdc_100174732606.0x000001

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Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing.

Authors: Nairy, Jayaprakash K.¹, Willoughby, Jennifer L. S.¹, Chan, Amy¹, Charisse, Klaus¹, Alam, Md. Rowshon¹, Qianfan Wang¹, Hoekstra, Menno², Kandasamy, Pachamuthu¹, Kel'in, Alexander V.¹, Milstein, Stuart¹, Taneja, Nate¹, O'Shea, Jonathan¹, Sarfraz Shaikh¹, Ligang Zhang¹, van der Sluis, Ronald J.², Jung, Michael E.³, Akinc, Akin¹, Hutabarat, Renta¹, Satya Kuchimanchi¹, Fitzgerald, Kevin¹

Source: Journal of the American Chemical Society; 12/10/2014, Vol. 136 Issue 49, p16958-16961, 4p

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siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivoin Hepatocytes

Authors: Matsuda, Shigeo, Keiser, Kristofer, Nair, Jayaprakash K., Charisse, Klaus, Manoharan, Rajar M., Kretschmer, Philip, Peng, Chang G., V. Kel’in, Alexander, Kandasamy, Pachamuthu, Willoughby, Jennifer L.S., Liebow, Abigail, Querbes, William, Yucius, Kristina, Nguyen, Tuyen, Milstein, Stuart, Maier, Martin A., Rajeev, Kallanthottathil G., Manoharan, Muthiah

Source: ACS Chemical Biology; May 2015, Vol. 10 Issue: 5 p1181-1187, 7p

Michael-Type Addition to 2-Nitrogalactal - A Simple Method to Access 1,1-Linked Oligosaccharides .

Authors: Rengarajan Balamurugan, Kandasamy Pachamuthu, Richard Schmidt

Source: Synlett; 2005, Vol. 2005 Issue 1, p134-138, 5p

Antibacterial Aminoglycosides with a Modified Mode of Binding to the Ribosomal-RNA Decoding SiteWe thank the NSERC and the Institut Universitaire de France for financial assistance provided to S.H. and E.W., respectively. Thanks are due to Lisa M. Risen for transcription/translation and MIC assays, and Dr. Kristen A. Sannes-Lowery for the KD determinations. We acknowledge the European Synchrotron Radiation Facility for provision of synchrotron radiation facilities, and we thank Stéphanie Monaco and Joanne McCarthy for assistance with the use of beamlines ID14-2 and ID14-4.

Authors: Boris François, Janek Szychowski, Susanta Sekhar Adhikari, Kandasamy Pachamuthu, Eric E. Swayze, Richard H. Griffey, Michael T. Migawa, Eric Westhof, Stephen Hanessian

Source: Angewandte Chemie International Edition; Dec2004, Vol. 43 Issue 48, p6735-6738, 4p

Synthesis of Methionine ContainingPeptides Related to Native Chemical Ligation.

Authors: Kandasamy Pachamuthu

Source: Synlett; Apr2003, Vol. 2003 Issue 5, p0659-0662, 4p