Search Results - "Firth, Mike"

In vivo CRISPR editing with no detectable genome-wide off-target mutations by Akcakaya, Pinar, Bobbin, Maggie L., Guo, Jimmy A., Malagon-Lopez, Jose, Clement, Kendell, Garcia, Sara P., Fellows, Mick D., Porritt, Michelle J., Firth, Mike A., Carreras, Alba, Baccega, Tania, Seeliger, Frank, Bjursell, Mikael, Tsai, Shengdar Q., Nguyen, Nhu T., Nitsch, Roberto, Mayr, Lorenz M., Pinello, Luca, Bohlooly-Y, Mohammad, Aryee, Martin J., Maresca, Marcello, Joung, J. Keith

“…CRISPR–Cas genome-editing nucleases hold substantial promise for developing human therapeutic applications 1 – 6 but identifying unwanted off-target mutations…”
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Pre-clinical pharmacology of AZD3965, a selective inhibitor of MCT1: DLBCL, NHL and Burkitt's lymphoma anti-tumor activity by Curtis, Nicola J, Mooney, Lorraine, Hopcroft, Lorna, Michopoulos, Filippos, Whalley, Nichola, Zhong, Haihong, Murray, Clare, Logie, Armelle, Revill, Mitchell, Byth, Kate F, Benjamin, Amanda D, Firth, Mike A, Green, Stephen, Smith, Paul D, Critchlow, Susan E

“…Tumors frequently display a glycolytic phenotype with increased flux through glycolysis and concomitant synthesis of lactate. To maintain glycolytic flux and…”
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CRISPR GUARD protects off-target sites from Cas9 nuclease activity using short guide RNAs by Coelho, Matthew A., De Braekeleer, Etienne, Firth, Mike, Bista, Michal, Lukasiak, Sebastian, Cuomo, Maria Emanuela, Taylor, Benjamin J. M.

“…Precise genome editing using CRISPR-Cas9 is a promising therapeutic avenue for genetic diseases, although off-target editing remains a significant safety…”
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Simultaneous inhibition of DNA-PK and Polϴ improves integration efficiency and precision of genome editing by Wimberger, Sandra, Akrap, Nina, Firth, Mike, Brengdahl, Johan, Engberg, Susanna, Schwinn, Marie K., Slater, Michael R., Lundin, Anders, Hsieh, Pei-Pei, Li, Songyuan, Cerboni, Silvia, Sumner, Jonathan, Bestas, Burcu, Schiffthaler, Bastian, Magnusson, Björn, Di Castro, Silvio, Iyer, Preeti, Bohlooly-Y, Mohammad, Machleidt, Thomas, Rees, Steve, Engkvist, Ola, Norris, Tyrell, Cadogan, Elaine B., Forment, Josep V., Šviković, Saša, Akcakaya, Pinar, Taheri-Ghahfarokhi, Amir, Maresca, Marcello

“…Genome editing, specifically CRISPR/Cas9 technology, has revolutionized biomedical research and offers potential cures for genetic diseases. Despite rapid…”
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Orthologue chemical space and its influence on target prediction by Mervin, Lewis H, Bulusu, Krishna C, Kalash, Leen, Afzal, Avid M, Svensson, Fredrik, Firth, Mike A, Barrett, Ian, Engkvist, Ola, Bender, Andreas

“…Abstract Motivation In silico approaches often fail to utilize bioactivity data available for orthologous targets due to insufficient evidence highlighting the…”
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BE-FLARE: a fluorescent reporter of base editing activity reveals editing characteristics of APOBEC3A and APOBEC3B by Coelho, Matthew A, Li, Songyuan, Pane, Luna Simona, Firth, Mike, Ciotta, Giovanni, Wrigley, Jonathan D, Cuomo, Maria Emanuela, Maresca, Marcello, Taylor, Benjamin J M

“…Base Editing is a precise genome editing method that uses a deaminase-Cas9 fusion protein to mutate cytidine to thymidine in target DNA in situ without the…”
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Targeted Metabolic Profiling of the Tg197 Mouse Model Reveals Itaconic Acid as a Marker of Rheumatoid Arthritis by Michopoulos, Filippos, Karagianni, Niki, Whalley, Nichola M, Firth, Mike A, Nikolaou, Christoforos, Wilson, Ian D, Critchlow, Susan E, Kollias, George, Theodoridis, Georgios A

“…Rheumatoid arthritis is a progressive, highly debilitating disease where early diagnosis, enabling rapid clinical intervention, would provide obvious benefits…”
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Harnessing DSB repair to promote efficient homology-dependent and -independent prime editing by Peterka, Martin, Akrap, Nina, Li, Songyuan, Wimberger, Sandra, Hsieh, Pei-Pei, Degtev, Dmitrii, Bestas, Burcu, Barr, Jack, van de Plassche, Stijn, Mendoza-Garcia, Patricia, Šviković, Saša, Sienski, Grzegorz, Firth, Mike, Maresca, Marcello

“…Prime editing recently emerged as a next-generation approach for precise genome editing. Here we exploit DNA double-strand break (DSB) repair to develop two…”
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Rapid target validation in a Cas9-inducible hiPSC derived kidney model by Shamshirgaran, Yasaman, Jonebring, Anna, Svensson, Anna, Leefa, Isabelle, Bohlooly-Y, Mohammad, Firth, Mike, Woollard, Kevin J., Hofherr, Alexis, Rogers, Ian M., Hicks, Ryan

“…Recent advances in induced pluripotent stem cells (iPSCs), genome editing technologies and 3D organoid model systems highlight opportunities to develop new in…”
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Understanding Cytotoxicity and Cytostaticity in a High-Throughput Screening Collection by Mervin, Lewis H, Cao, Qing, Barrett, Ian P, Firth, Mike A, Murray, David, McWilliams, Lisa, Haddrick, Malcolm, Wigglesworth, Mark, Engkvist, Ola, Bender, Andreas

“…While mechanisms of cytotoxicity and cytostaticity have been studied extensively from the biological side, relatively little is currently understood regarding…”
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Engineered Cas9 extracellular vesicles as a novel gene editing tool by Osteikoetxea, Xabier, Silva, Andreia, Lázaro‐Ibáñez, Elisa, Salmond, Nikki, Shatnyeva, Olga, Stein, Josia, Schick, Jan, Wren, Stephen, Lindgren, Julia, Firth, Mike, Madsen, Alexandra, Mayr, Lorenz M., Overman, Ross, Davies, Rick, Dekker, Niek

“…Extracellular vesicles (EVs) have shown promise as biological delivery vehicles, but therapeutic applications require efficient cargo loading. Here, we…”
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In vivo phage display identifies novel peptides for cardiac targeting by Ivanova, Alena, Kohl, Franziska, González-King Garibotti, Hernán, Chalupska, Renata, Cvjetkovic, Aleksander, Firth, Mike, Jennbacken, Karin, Martinsson, Sofia, Silva, Andreia M., Viken, Ida, Wang, Qing-Dong, Wiseman, John, Dekker, Niek

“…Heart failure remains a leading cause of mortality. Therapeutic intervention for heart failure would benefit from targeted delivery to the damaged heart…”
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Epigenetic modulators link mitochondrial redox homeostasis to cardiac function in a sex-dependent manner by ElBeck, Zaher, Hossain, Mohammad Bakhtiar, Siga, Humam, Oskolkov, Nikolay, Karlsson, Fredrik, Lindgren, Julia, Walentinsson, Anna, Koppenhöfer, Dominique, Jarvis, Rebecca, Bürli, Roland, Jamier, Tanguy, Franssen, Elske, Firth, Mike, Degasperi, Andrea, Bendtsen, Claus, Menzies, Robert I., Streckfuss-Bömeke, Katrin, Kohlhaas, Michael, Nickel, Alexander G., Lund, Lars H., Maack, Christoph, Végvári, Ákos, Betsholtz, Christer

“…While excessive production of reactive oxygen species (ROS) is a characteristic hallmark of numerous diseases, clinical approaches that ameliorate oxidative…”
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A Type II-B Cas9 nuclease with minimized off-targets and reduced chromosomal translocations in vivo by Bestas, Burcu, Wimberger, Sandra, Degtev, Dmitrii, Madsen, Alexandra, Rottner, Antje K., Karlsson, Fredrik, Naumenko, Sergey, Callahan, Megan, Touza, Julia Liz, Francescatto, Margherita, Möller, Carl Ivar, Badertscher, Lukas, Li, Songyuan, Cerboni, Silvia, Selfjord, Niklas, Ericson, Elke, Gordon, Euan, Firth, Mike, Chylinski, Krzysztof, Taheri-Ghahfarokhi, Amir, Bohlooly-Y, Mohammad, Snowden, Mike, Pangalos, Menelaos, Nuttall, Barrett, Akcakaya, Pinar, Sienski, Grzegorz, Maresca, Marcello

“…Streptococcus pyogenes Cas9 (SpCas9) and derived enzymes are widely used as genome editors, but their promiscuous nuclease activity often induces undesired…”
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Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes by Zabulica, Mihaela, Srinivasan, Raghuraman C., Akcakaya, Pinar, Allegri, Gabriella, Bestas, Burcu, Firth, Mike, Hammarstedt, Christina, Jakobsson, Tomas, Jakobsson, Towe, Ellis, Ewa, Jorns, Carl, Makris, Georgios, Scherer, Tanja, Rimann, Nicole, van Zuydam, Natalie R., Gramignoli, Roberto, Forslöw, Anna, Engberg, Susanna, Maresca, Marcello, Rooyackers, Olav, Thöny, Beat, Häberle, Johannes, Rosen, Barry, Strom, Stephen C.

“…Ornithine transcarbamylase deficiency (OTCD) is a monogenic disease of ammonia metabolism in hepatocytes. Severe disease is frequently treated by orthotopic…”
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Author Correction: Universal toxin-based selection for precise genome engineering in human cells by Li, Songyuan, Akrap, Nina, Cerboni, Silvia, Porritt, Michelle J., Wimberger, Sandra, Lundin, Anders, Möller, Carl, Firth, Mike, Gordon, Euan, Lazovic, Bojana, Sieńska, Aleksandra, Pane, Luna Simona, Coelho, Matthew A., Ciotta, Giovanni, Pellegrini, Giovanni, Sini, Marcella, Xu, Xiufeng, Mitra, Suman, Bohlooly-Y, Mohammad, Taylor, Benjamin J. M., Sienski, Grzegorz, Maresca, Marcello

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An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation by Porreca, Immacolata, Blassberg, Robert, Harbottle, Jennifer, Joubert, Bronwyn, Mielczarek, Olga, Stombaugh, Jesse, Hemphill, Kevin, Sumner, Jonathan, Pazeraitis, Deividas, Touza, Julia Liz, Francescatto, Margherita, Firth, Mike, Selmi, Tommaso, Collantes, Juan Carlos, Strezoska, Zaklina, Taylor, Benjamin, Jin, Shengkan, Wiggins, Ceri M, van Brabant Smith, Anja, Lambourne, John J

“…Gene editing technologies hold promise for enabling the next generation of adoptive cellular therapies. In conventional gene editing platforms that rely on…”
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Universal toxin-based selection for precise genome engineering in human cells by Li, Songyuan, Akrap, Nina, Cerboni, Silvia, Porritt, Michelle J., Wimberger, Sandra, Lundin, Anders, Möller, Carl, Firth, Mike, Gordon, Euan, Lazovic, Bojana, Sieńska, Aleksandra, Pane, Luna Simona, Coelho, Matthew A., Ciotta, Giovanni, Pellegrini, Giovanni, Sini, Marcella, Xu, Xiufeng, Mitra, Suman, Bohlooly-Y, Mohammad, Taylor, Benjamin J. M., Sienski, Grzegorz, Maresca, Marcello

“…Prokaryotic restriction enzymes, recombinases and Cas proteins are powerful DNA engineering and genome editing tools. However, in many primary cell types, the…”
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Systematic Evaluation of CRISPRa and CRISPRi Modalities Enables Development of a Multiplexed, Orthogonal Gene Activation and Repression System by Martella, Andrea, Firth, Mike, Taylor, Benjamin J. M, Göppert, Anne, Cuomo, Emanuela M, Roth, Robert G, Dickson, Alan J, Fisher, David I

“…The ability to manipulate the expression of mammalian genes using synthetic transcription factors is highly desirable in both fields of basic research and…”
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High-content phenotypic assay for proliferation of human iPSC-derived cardiomyocytes identifies L-type calcium channels as targets by Woo, Laura A., Tkachenko, Svyatoslav, Ding, Mei, Plowright, Alleyn T., Engkvist, Ola, Andersson, Henrik, Drowley, Lauren, Barrett, Ian, Firth, Mike, Akerblad, Peter, Wolf, Matthew J., Bekiranov, Stefan, Brautigan, David L., Wang, Qing-Dong, Saucerman, Jeffrey J.

“…Over 5 million people in the United States suffer from heart failure, due to the limited ability to regenerate functional cardiac tissue. One potential…”
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