A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene von Kemaladewi, Dwi U., Bassi, Prabhpreet S., Erwood, Steven, Al-Basha, Dhekra, Gawlik, Kinga I., Lindsay, Kyle, Hyatt, Elzbieta, Kember, Rebekah, Place, Kara M., Marks, Ryan M., Durbeej, Madeleine, Prescott, Steven A., Ivakine, Evgueni A., Cohn, Ronald D.

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Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders von Wojtal, Daria, Kemaladewi, Dwi U., Malam, Zeenat, Abdullah, Sarah, Wong, Tatianna W.Y., Hyatt, Elzbieta, Baghestani, Zahra, Pereira, Sergio, Stavropoulos, James, Mouly, Vincent, Mamchaoui, Kamel, Muntoni, Francesco, Voit, Thomas, Gonorazky, Hernan D., Dowling, James J., Wilson, Michael D., Mendoza-Londono, Roberto, Ivakine, Evgueni A., Cohn, Ronald D.

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Saturation variant interpretation using CRISPR prime editing von Erwood, Steven, Bily, Teija M. I., Lequyer, Jason, Yan, Joyce, Gulati, Nitya, Brewer, Reid A., Zhou, Liangchi, Pelletier, Laurence, Ivakine, Evgueni A., Cohn, Ronald D.

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An optimized toolkit for prime editing von Marks, Ryan M., Scott, Ori, Ivakine, Evgueni A., Cohn, Ronald D.

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Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies von Fatehi, Sina, Marks, Ryan M, Rok, Matthew J, Perillat, Lucie, Ivakine, Evgueni A, Cohn, Ronald D

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The human Stat1 gain-of-function T385M mutation causes expansion of activated T-follicular helper/T-helper 1-like CD4 T cells and sex-biased autoimmunity in specific pathogen-free... von Scott, Ori, Visuvanathan, Shagana, Reddy, Emily, Mahamed, Deeqa, Gu, Bin, Roifman, Chaim M, Cohn, Ronald D, Guidos, Cynthia J, Ivakine, Evgueni A

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Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism von Kemaladewi, Dwi U, Maino, Eleonora, Hyatt, Elzbieta, Hou, Huayun, Ding, Maylynn, Place, Kara M, Zhu, Xinyi, Bassi, Prabhpreet, Baghestani, Zahra, Deshwar, Amit G, Merico, Daniele, Xiong, Hui Y, Frey, Brendan J, Wilson, Michael D, Ivakine, Evgueni A, Cohn, Ronald D

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Kainate receptor auxiliary subunit NETO2 is required for normal fear expression and extinction von Mennesson, Marie, Rydgren, Emilie, Lipina, Tatiana, Sokolowska, Ewa, Kulesskaya, Natalia, Morello, Francesca, Ivakine, Evgueni, Voikar, Vootele, Risbrough, Victoria, Partanen, Juha, Hovatta, Iiris

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Efficient prime editing in two-cell mouse embryos using PEmbryo von Kim-Yip, Rebecca P., McNulty, Ryan, Joyce, Bradley, Mollica, Antonio, Chen, Peter J., Ravisankar, Purnima, Law, Benjamin K., Liu, David R., Toettcher, Jared E., Ivakine, Evgueni A., Posfai, Eszter, Adamson, Britt

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Kainate Receptors Coexist in a Functional Complex with KCC2 and Regulate Chloride Homeostasis in Hippocampal Neurons von Mahadevan, Vivek, Pressey, Jessica C., Acton, Brooke A., Uvarov, Pavel, Huang, Michelle Y., Chevrier, Jonah, Puchalski, Andrew, Li, Caiwei M., Ivakine, Evgueni A., Airaksinen, Matti S., Delpire, Eric, McInnes, Roderick R., Woodin, Melanie A.

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Saturation genome editing maps the functional spectrum of pathogenic VHL alleles von Buckley, Megan, Terwagne, Chloé, Ganner, Athina, Cubitt, Laura, Brewer, Reid, Kim, Dong-Kyu, Kajba, Christina M., Forrester, Nicole, Dace, Phoebe, De Jonghe, Joachim, Shepherd, Scott T. C., Sawyer, Chelsea, McEwen, Mairead, Diederichs, Sven, Neumann-Haefelin, Elke, Turajlic, Samra, Ivakine, Evgueni A., Findlay, Gregory M.

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Multi-gene duplication removal in an engineered human cellular MECP2 duplication syndrome model with an IRAK1-MECP2 duplication von Rizvi, Samar Z., Chan, Wing Suen, Maino, Eleonora, Steiman, Sydney, Forguson, Georgiana, Klepfish, Maya, Cohn, Ronald D., Ivakine, Evgueni A.

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Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression von Maino, Eleonora, Wojtal, Daria, Evagelou, Sonia L, Farheen, Aiman, Wong, Tatianna W Y, Lindsay, Kyle, Scott, Ori, Rizvi, Samar Z, Hyatt, Elzbieta, Rok, Matthew, Visuvanathan, Shagana, Chiodo, Amanda, Schneeweiss, Michelle, Ivakine, Evgueni A, Cohn, Ronald D

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Modeling Niemann-Pick disease type C in a human haploid cell line allows for patient variant characterization and clinical interpretation von Erwood, Steven, Brewer, Reid A, Bily, Teija M I, Maino, Eleonora, Zhou, Liangchi, Cohn, Ronald D, Ivakine, Evgueni A

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A new mouse model of ATR-X syndrome carrying a common patient mutation exhibits neurological and morphological defects von Tillotson, Rebekah, Yan, Keqin, Ruston, Julie, DeYoung, Taylor, Córdova, Alex, Turcotte-Cardin, Valérie, Yee, Yohan, Taylor, Christine, Visuvanathan, Shagana, Babbs, Christian, Ivakine, Evgueni A, Sled, John G, Nieman, Brian J, Picketts, David J, Justice, Monica J

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A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy von Wong, Tatianna Wai Ying, Ahmed, Abdalla, Yang, Grace, Maino, Eleonora, Steiman, Sydney, Hyatt, Elzbieta, Chan, Parry, Lindsay, Kyle, Wong, Nicole, Golebiowski, Diane, Schneider, Joel, Delgado-Olguín, Paul, Ivakine, Evgueni A, Cohn, Ronald D

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Maintaining skeletal muscle mass: lessons learned from hibernation von Ivakine, Evgueni A., Cohn, Ronald D.

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Neto2 is a KCC2 interacting protein required for neuronal Cl⁻ regulation in hippocampal neurons von Ivakine, Evgueni A., Acton, Brooke A., Mahadevan, Vivek, Ormond, Jake, Tang, Man, Pressey, Jessica C., Huang, Michelle Y., Ng, David, Delpire, Eric, Salter, Michael W., Woodin, Melanie A., McInnes, Roderick R.

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An Irak1-Mecp2 tandem duplication mouse model for the study of MECP2 duplication syndrome von Maino, Eleonora, Scott, Ori, Rizvi, Samar Z, Chan, Wing Suen, Visuvanathan, Shagana, Zablah, Youssif Ben, Li, Hongbin, Sengar, Ameet S, Salter, Michael W, Jia, Zhengping, Rossant, Janet, Cohn, Ronald D, Gu, Bin, Ivakine, Evgueni A

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Generation and characterization of a novel mouse model of Becker Muscular Dystrophy with a deletion of exons 52 to 55 von Perillat, Lucie O M, Wong, Tatianna W Y, Maino, Eleonora, Ahmed, Abdalla, Scott, Ori, Hyatt, Elzbieta, Delgado-Olguin, Paul, Visuvanathan, Shagana, Ivakine, Evgueni A, Cohn, Ronald D

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