Anastasia Khvorova

Publication Activity (10 Years)

Years Active: 2018-2024
Publications (10 Years): 57

Top Topics

Duchenne Muscular Dystrophy

Blood Brain Barrier

Top Venues

Nucleic acids research

bioRxiv : the preprint server for biology

Nature reviews. Drug discovery

Nature biotechnology

This page only lists publications with an associated author ORCID identifier.

Publications

Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. eLife 12 (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery (2024)
Alexandra Weiss, James W Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal M Ferguson, Elif O Dogan, Nicholas Wightman, Kit Mocarski, Dimas Echeverria Moreno, Ashley Summers, Brianna Bramato, Nicholas McHugh, Raymond Furgal, Nozomi Yamada, David Cooper, Kathryn R Monopoli, Bruno M D C Godinho, Matthew R Hassler, Ken Yamada, Paul Greer, Nils Henninger, Robert H Brown, Anastasia Khvorova
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice. bioRxiv : the preprint server for biology (2024)
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. eLife 12 (2024)
Denis Susorov, Dimas Echeverria Moreno, Anastasia Khvorova, Andrei A Korostelev
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic acids research 52 (15) (2024)
Jillian Belgrad, Anastasia Khvorova
More than 185 CAG repeats: a point of no return in Huntington's disease biology. Brain : a journal of neurology 147 (5) (2024)
Ken Yamada, Vignesh Narayan Hariharan, Jillian Caiazzi, Rachael Miller, Chantal M Ferguson, Ellen Sapp, Hassan H Fakih, Qi Tang, Nozomi Yamada, Raymond C Furgal, Joseph D Paquette, Annabelle Biscans, Brianna M Bramato, Nicholas McHugh, Ashley Summers, Clemens Lochmann, Bruno M D C Godinho, Samuel Hildebrand, Samuel O Jackson, Dimas Echeverria, Matthew R Hassler, Julia F Alterman, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones. Nature biotechnology (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research 52 (11) (2024)
Denis Susorov, Dimas Echeverria Moreno, Anastasia Khvorova, Andrei A Korostelev
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic acids research (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research 52 (11) (2024)
Chantal M Ferguson, Bruno M D C Godinho, Dimas Echeverria Moreno, Matthew R Hassler, Lorenc Vangjeli, Jacquelyn Sousa, Nicholas McHugh, Julia F Alterman, Vignesh Narayan Hariharan, Pranathi Meda Krishnamurthy, Jonathan K Watts, Eveny Rogaev, Anastasia Khvorova
A combinatorial approach for achieving CNS-selective RNAi. Nucleic acids research 52 (9) (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo . bioRxiv : the preprint server for biology (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research 52 (11) (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research (2024)
Ken Yamada, Vignesh Narayan Hariharan, Jillian Caiazzi, Rachael Miller, Chantal M Ferguson, Ellen Sapp, Hassan H Fakih, Qi Tang, Nozomi Yamada, Raymond C Furgal, Joseph D Paquette, Annabelle Biscans, Brianna M Bramato, Nicholas McHugh, Ashley Summers, Clemens Lochmann, Bruno M D C Godinho, Samuel Hildebrand, Samuel O Jackson, Dimas Echeverria, Matthew R Hassler, Julia F Alterman, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones. Nature biotechnology (2024)
Chantal M Ferguson, Bruno M D C Godinho, Dimas Echeverria Moreno, Matthew R Hassler, Lorenc Vangjeli, Jacquelyn Sousa, Nicholas McHugh, Julia F Alterman, Vignesh Narayan Hariharan, Pranathi Meda Krishnamurthy, Jonathan K Watts, Eveny Rogaev, Anastasia Khvorova
A combinatorial approach for achieving CNS-selective RNAi. Nucleic acids research 52 (9) (2024)
Jillian Belgrad, Anastasia Khvorova
More than 185 CAG repeats: a point of no return in Huntington's disease biology. Brain : a journal of neurology (2024)
Alexandra Weiss, James W Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal M Ferguson, Elif O Dogan, Nicholas Wightman, Kit Mocarski, Dimas Echeverria Moreno, Ashley Summers, Brianna Bramato, Nicholas McHugh, Raymond Furgal, Nozomi Yamada, David Cooper, Kathryn R Monopoli, Bruno M D C Godinho, Matthew R Hassler, Ken Yamada, Paul L Greer, Nils Henninger, Robert H Brown, Anastasia Khvorova
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice. bioRxiv : the preprint server for biology (2024)
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. eLife 12 (2024)
Denis Susorov, Dimas Echeverria Moreno, Anastasia Khvorova, Andrei A Korostelev
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic acids research 52 (15) (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery 23 (5) (2024)
Chantal M Ferguson, Bruno M D C Godinho, Dimas Echeverria Moreno, Matthew R Hassler, Lorenc Vangjeli, Jacquelyn Sousa, Nicholas McHugh, Julia F Alterman, Vignesh Narayan Hariharan, Pranathi Meda Krishnamurthy, Jonathan K Watts, Eveny Rogaev, Anastasia Khvorova
A combinatorial approach for achieving CNS-selective RNAi. Nucleic acids research 52 (9) (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo . bioRxiv : the preprint server for biology (2024)
Denis Susorov, Dimas Echeverria Moreno, Anastasia Khvorova, Andrei A Korostelev
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic acids research 52 (15) (2024)
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Hui Wang, Nicholas McHugh, Brianna Bramato, Brett Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. eLife 12 (2024)
Chantal M Ferguson, Bruno M D C Godinho, Dimas Echeverria Moreno, Matthew R Hassler, Lorenc Vangjeli, Jacquelyn Sousa, Nicholas McHugh, Julia F Alterman, Vignesh Narayan Hariharan, Pranathi Meda Krishnamurthy, Jonathan K Watts, Eveny Rogaev, Anastasia Khvorova
A combinatorial approach for achieving CNS-selective RNAi. Nucleic acids research 52 (9) (2024)
Jillian Belgrad, Qi Tang, Samuel R Hildebrand, Ashley Summers, Ellen Sapp, Dimas Echeverria Moreno, Daniel O'Reilly, Eric Luu, Brianna Bramato, Sarah Allen, David Cooper, Julia F Alterman, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic acids research (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo . bioRxiv : the preprint server for biology (2024)
Jillian Belgrad, Anastasia Khvorova
More than 185 CAG repeats: a point of no return in Huntington's disease biology. Brain : a journal of neurology 147 (5) (2024)
Chantal M Ferguson, Bruno M D C Godinho, Dimas Echeverria Moreno, Matthew R Hassler, Lorenc Vangjeli, Jacquelyn Sousa, Nicholas McHugh, Julia F Alterman, Vignesh Narayan Hariharan, Pranathi Meda Krishnamurthy, Jonathan K Watts, Eveny Rogaev, Anastasia Khvorova
A combinatorial approach for achieving CNS-selective RNAi. Nucleic acids research (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery 23 (5) (2024)
Alexandra Weiss, James W Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal M Ferguson, Elif O Dogan, Nicholas Wightman, Kit Mocarski, Dimas Echeverria Moreno, Ashley Summers, Brianna Bramato, Nicholas McHugh, Raymond Furgal, Nozomi Yamada, David Cooper, Kathryn R Monopoli, Bruno M D C Godinho, Matthew R Hassler, Ken Yamada, Paul L Greer, Nils Henninger, Robert H Brown, Anastasia Khvorova
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice. bioRxiv : the preprint server for biology (2024)
Rachael Miller, Joseph Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna Bramato, Nozomi Yamada, Julia F Alterman, Dimas Echeveria, Ken Yamada, Jonathan K Watts, Christelle Anaclet, Marian DiFiglia, Anastasia Khvorova, Neil Aronin
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca 2+ and Mg 2+ in the formulation. bioRxiv : the preprint server for biology (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo . bioRxiv : the preprint server for biology (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo . bioRxiv : the preprint server for biology (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery 23 (5) (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery 23 (5) (2024)
Qi Tang, Anastasia Khvorova
RNAi-based drug design: considerations and future directions. Nature reviews. Drug discovery 23 (5) (2024)
Rachael Miller, Joseph Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna Bramato, Nazomi Yamada, Julia F Alterman, Dimas Echeveria, Ken Yamada, Jonathan K Watts, Christelle Anaclet, Marian DiFiglia, Anastasia Khvorova, Neil Aronin
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation. bioRxiv : the preprint server for biology (2024)
Denis Susorov, Dimas Echeverria Moreno, Anastasia Khvorova, Andrei A Korostelev
mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic acids research 52 (15) (2024)
Vignesh Narayan Hariharan, Jillian Caiazzi, Rachael Miller, Chantal M Ferguson, Ellen Sapp, Hassan Fakih, Qi Tang, Nozomi Yamada, Raymond Furgal, Joseph Paquette, Brianna Bramato, Nicholas McHugh, Ashley Summers, Clemens Lochmann, Bruno M D C Godinho, Samuel Hildebrand, Dimas Echeverria, Matthew R Hassler, Julia F Alterman, Marian DiFiglia, Neil Aronin, Anastasia Khvorova, Ken Yamada
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo. Research square (2023)
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria Moreno, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Nicholas McHugh, Brianna Bramato, Brett M Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. bioRxiv : the preprint server for biology (2023)
Han Zhang, Karen Kelly, Jonathan Lee, Dimas Echeverria Moreno, David A Cooper, Rebecca Panwala, Zexiang Chen, Nicholas Gaston, Gregory A Newby, Jun Xie, David R Liu, Guangping Gao, Scot A Wolfe, Anastasia Khvorova, Jonathan K Watts, Erik J Sontheimer
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo . bioRxiv : the preprint server for biology (2023)
Samuel R Hildebrand, Adam K Hedger, Jonathan K Watts, Anastasia Khvorova
Formamide significantly enhances the efficiency of chemical adenylation of RNA sequencing ligation adaptors. RNA (New York, N.Y.) (2023)
Qi Tang, Hassan H Fakih, Mohammad Zain Ui Abideen, Samuel R Hildebrand, Khashayar Afshari, Katherine Y Gross, Jacquelyn Sousa, Allison S Maebius, Christina Bartholdy, Pia Pernille Søgaard, Malene Jackerott, Vignesh Narayan Hariharan, Ashley Summers, Xueli Fan, Ken Okamura, Kathryn R Monopoli, David A Cooper, Dimas Echeverria, Brianna Bramato, Nicholas McHugh, Raymond C Furgal, Karen Dresser, Sarah J Winter, Annabelle Biscans, Jane Chuprin, Nazgol-Sadat Haddadi, Shany Sherman, Ümmügülsüm Yıldız-Altay, Mehdi Rashighi, Jillian M Richmond, Claire Bouix-Peter, Carine Blanchard, Adam Clauss, Julia F Alterman, Anastasia Khvorova, John E Harris
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin. Nature communications 14 (1) (2023)
Han Zhang, Karen Kelly, Jonathan Lee, Dimas Echeverria Moreno, David Cooper, Rebecca Panwala, Nadia Amrani, Zexiang Chen, Nicholas Gaston, Atish Wagh, Gregory A Newby, Jun Xie, David R Liu, Guangping Gao, Scot A Wolfe, Anastasia Khvorova, Jonathan K Watts, Erik J Sontheimer
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Nucleic acids research (2023)
Kyounghee Min, Batuhan Yenilmez, Mark Kelly, Dimas Echeverria Moreno, Michael Elleby, Lawrence M Lifshitz, Naideline Raymond, Emmanouela Tsagkaraki, Shauna M Harney, Chloe DiMarzio, Nicholas McHugh, Brianna Bramato, Brett M Morrison, Jeffery D Rothstein, Anastasia Khvorova, Michael P Czech
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis. bioRxiv : the preprint server for biology (2023)
Ken Yamada, Samuel R Hildebrand, Sarah M Davis, Rachael Miller, Faith Conroy, Ellen Sapp, Jillian Caiazzi, Julia F Alterman, Loïc Roux, Dimas Echeverria Moreno, Matthew R Hassler, Edith L Pfister, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity. Nucleic acids research 49 (21) (2022)
Faith Conroy, Rachael Miller, Julia F Alterman, Matthew R Hassler, Dimas Echeverria, Bruno M D C Godinho, Emily G Knox, Ellen Sapp, Jaquelyn Sousa, Ken Yamada, Farah Mahmood, Adel Boudi, Kimberly Kegel-Gleason, Marian DiFiglia, Neil Aronin, Anastasia Khvorova, Edith L Pfister
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models. Nature communications 13 (1) (2022)
Socheata Ly, Marie-Cécile Didiot, Chantal M Ferguson, Andrew H Coles, Rachael Miller, Kathryn Chase, Dimas Echeverria Moreno, Feng Wang, Ghazaleh Sadri-Vakili, Neil Aronin, Anastasia Khvorova
Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains. Brain communications 4 (6) (2022)
Minwook Shin, Io Long Chan, Yuming Cao, Alisha M Gruntman, Jonathan Lee, Jacquelyn Sousa, Tomás C Rodríguez, Dimas Echeverria Moreno, Gitali Devi, Alexandre J Debacker, Michael P Moazami, Pranathi Meda Krishnamurthy, Julia M Rembetsy-Brown, Karen Kelly, Onur Yukselen, Elisa Donnard, Teagan J Parsons, Anastasia Khvorova, Erik J Sontheimer, René Maehr, Manuel Garber, Jonathan K Watts
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts. Nucleic acids research 50 (15) (2022)
Krishanu Saha, Erik J Sontheimer, P J Brooks, Melinda R Dwinell, Charles A Gersbach, David R Liu, Stephen A Murray, Shengdar Q Tsai, Ross C Wilson, Daniel G Anderson, Aravind Asokan, Jillian F Banfield, Krystof S Bankiewicz, Gang Bao, Jeff W M Bulte, Nenad Bursac, Jarryd M Campbell, Daniel F Carlson, Elliot L Chaikof, Zheng-Yi Chen, R Holland Cheng, Karl J Clark, David T Curiel, James E Dahlman, Benjamin E Deverman, Mary E Dickinson, Jennifer A Doudna, Stephen C Ekker, Marina E Emborg, Guoping Feng, Benjamin S Freedman, David M Gamm, Guangping Gao, Ionita C Ghiran, Peter M Glazer, Shaoqin Gong, Jason D Heaney, Jon D Hennebold, John T Hinson, Anastasia Khvorova, Samira Kiani, William R Lagor, Kit S Lam, Kam W Leong, Jon E Levine, Jennifer A Lewis, Cathleen M Lutz, Danith H Ly, Samantha Maragh, Paul B McCray, Todd C McDevitt, Oleg Mirochnitchenko, Ryuji Morizane, Niren Murthy, Randall S Prather, John A Ronald, Subhojit Roy, Sushmita Roy, Venkata Sabbisetti, W Mark Saltzman, Philip J Santangelo, David J Segal, Mary Shimoyama, Melissa C Skala, Alice F Tarantal, John C Tilton, George A Truskey, Moriel Vandsburger, Jonathan K Watts, Kevin D Wells, Scot A Wolfe, Qiaobing Xu, Wen Xue, Guohua Yi, Jiangbing Zhou, null null
The NIH Somatic Cell Genome Editing program. Nature 592 (7853) (2021)
Keitaro Fukuda, Ken Okamura, Rebecca L Riding, Xueli Fan, Khashayar Afshari, Nazgol-Sadat Haddadi, Sean Matthew McCauley, Mehmet Hakan Guney, Jeremy Luban, Takeru Funakoshi, Tomonori Yaguchi, Yutaka Kawakami, Anastasia Khvorova, Katherine A Fitzgerald, John E Harris
AIM2 regulates anti-tumor immunity and is a viable therapeutic target for melanoma. The Journal of experimental medicine 218 (9) (2021)
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 L Gray-Edwards, Richard P Moser, Nina C Bishop, Samer M Jaber, Matthew J Gounis, Miguel Sena-Esteves, Athma A Pai, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system. Nature biotechnology 37 (8) (2019)
Anton A Turanov, Agnes Lo, Matthew R Hassler, Angela Makris, Ami Ashar-Patel, Julia F Alterman, Andrew H Coles, Reka A Haraszti, Loic Roux, Bruno M D C Godinho, Dimas Echeverria, Suzanne Pears, Jim Iliopoulos, Renuka Shanmugalingam, Robert Ogle, Zsuzsanna K Zsengeller, Annemarie Hennessy, S Ananth Karumanchi, Melissa J Moore, Anastasia Khvorova
RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Nature biotechnology (2018)