Zobrazeno 1 - 10
of 209
pro vyhledávání: '"J. Sontheimer"'
Autor:
Nathan Bamidele, Han Zhang, Xiaolong Dong, Haoyang Cheng, Nicholas Gaston, Hailey Feinzig, Hanbing Cao, Karen Kelly, Jonathan K. Watts, Jun Xie, Guangping Gao, Erik J. Sontheimer
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-13 (2024)
Abstract Nme2Cas9 has been established as a genome editing platform with compact size, high accuracy, and broad targeting range, including single-AAV-deliverable adenine base editors. Here, we engineer Nme2Cas9 to further increase the activity and ta
Externí odkaz:
https://doaj.org/article/df3d02ad296b4505893c699918456904
Autor:
Lin Zhao, Sabrina R. T. Koseki, Rachel A. Silverstein, Nadia Amrani, Christina Peng, Christian Kramme, Natasha Savic, Martin Pacesa, Tomás C. Rodríguez, Teodora Stan, Emma Tysinger, Lauren Hong, Vivian Yudistyra, Manvitha R. Ponnapati, Joseph M. Jacobson, George M. Church, Noah Jakimo, Ray Truant, Martin Jinek, Benjamin P. Kleinstiver, Erik J. Sontheimer, Pranam Chatterjee
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-8 (2023)
Abstract CRISPR enzymes require a defined protospacer adjacent motif (PAM) flanking a guide RNA-programmed target site, limiting their sequence accessibility for robust genome editing applications. In this study, we recombine the PAM-interacting doma
Externí odkaz:
https://doaj.org/article/c6eaa1afc2b34657a91e037a58ba1052
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-9 (2023)
Abstract Targeted insertion of large DNA fragments holds promise for genome engineering and gene therapy. Prime editing (PE) effectively inserts short (400 bp) remains low and in vivo application has not been demonstrated. Inspired by the efficient g
Externí odkaz:
https://doaj.org/article/198f86c79bc94a7cb8636247cdf4344c
Autor:
Katharina E. Meijboom, Abbas Abdallah, Nicholas P. Fordham, Hiroko Nagase, Tomás Rodriguez, Carolyn Kraus, Tania F. Gendron, Gopinath Krishnan, Rustam Esanov, Nadja S. Andrade, Matthew J. Rybin, Melina Ramic, Zachary D. Stephens, Alireza Edraki, Meghan T. Blackwood, Aydan Kahriman, Nils Henninger, Jean-Pierre A. Kocher, Michael Benatar, Michael H. Brodsky, Leonard Petrucelli, Fen-Biao Gao, Erik J. Sontheimer, Robert H. Brown, Zane Zeier, Christian Mueller
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-17 (2022)
A hexanucleotide repeat expansion in C9ORF72 is the most common genetic cause of ALS and FTD. Here, the authors demonstrate CRISPR/Cas9 excision of the expansion results in a rescue of disease mechanisms in vivo and in vitro.
Externí odkaz:
https://doaj.org/article/3c12d94acb99423da250fefe20d4cde2
Autor:
Shun-Qing Liang, Pengpeng Liu, Jordan L. Smith, Esther Mintzer, Stacy Maitland, Xiaolong Dong, Qiyuan Yang, Jonathan Lee, Cole M. Haynes, Lihua Julie Zhu, Jonathan K. Watts, Erik J. Sontheimer, Scot A. Wolfe, Wen Xue
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
In vivo assessment of nuclease off-target activity has primarily been indirect or through ChIP-based detection of double-strand break DNA repair factors, which can be cumbersome. Here, the authors show that GUIDE-tag, enables one-step off-target geno
Externí odkaz:
https://doaj.org/article/102ac89073204ae399bf4e3bc3f7fb8c
Autor:
Raed Ibraheim, Phillip W. L. Tai, Aamir Mir, Nida Javeed, Jiaming Wang, Tomás C. Rodríguez, Suk Namkung, Samantha Nelson, Eraj Shafiq Khokhar, Esther Mintzer, Stacy Maitland, Zexiang Chen, Yueying Cao, Emmanouela Tsagkaraki, Scot A. Wolfe, Dan Wang, Athma A. Pai, Wen Xue, Guangping Gao, Erik J. Sontheimer
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
Long-term expression of Cas9 following precision genome editing in vivo may lead to undesirable consequences. Here we show that a single-vector, self-inactivating AAV system containing Cas9 nuclease, guide, and DNA donor can use homology-directed rep
Externí odkaz:
https://doaj.org/article/81e517a4261d442a884395258d491bfe
Autor:
Emmanouela Tsagkaraki, Sarah M. Nicoloro, Tiffany DeSouza, Javier Solivan-Rivera, Anand Desai, Lawrence M. Lifshitz, Yuefei Shen, Mark Kelly, Adilson Guilherme, Felipe Henriques, Nadia Amrani, Raed Ibraheim, Tomas C. Rodriguez, Kevin Luk, Stacy Maitland, Randall H. Friedline, Lauren Tauer, Xiaodi Hu, Jason K. Kim, Scot A. Wolfe, Erik J. Sontheimer, Silvia Corvera, Michael P. Czech
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
Worldwide pandemics of obesity and diabetes prompt an urgent need for new approaches to their prevention and cure. Here the authors present a CRISPR-based strategy that enhances the therapeutic potential of human adipocytes when implanted in obese mi
Externí odkaz:
https://doaj.org/article/9db95209ec9c4296b7f96552abbf9db0
Autor:
Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, Wen Xue
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-13 (2021)
Prime editors use a template sequence within their pegRNA to facilitate nucleotide substitutions or local indels. Here the authors use AAVs to deliver a split-intein prime editor in vivo to correct a pathogenic mutation.
Externí odkaz:
https://doaj.org/article/f79d7d69ebf14bc9b101910fdc770c8c
Autor:
Pranam Chatterjee, Jooyoung Lee, Lisa Nip, Sabrina R. T. Koseki, Emma Tysinger, Erik J. Sontheimer, Joseph M. Jacobson, Noah Jakimo
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-6 (2020)
Protospacer adjacent motif (PAM) requirements limit the target range of CRISPR endonucleases. Here, the authors graft the 5$$^{\prime}$$ ′ -NAAN-3$$^{\prime}$$ ′ PAM-interacting domain of SmacCas9 onto SpyCas9 to create adenine dinucleotide targe
Externí odkaz:
https://doaj.org/article/de6c445336ca4aacb34b61cb8daeb358
Autor:
Krishna S Ghanta, Zexiang Chen, Aamir Mir, Gregoriy A Dokshin, Pranathi M Krishnamurthy, Yeonsoo Yoon, Judith Gallant, Ping Xu, Xiao-Ou Zhang, Ahmet Rasit Ozturk, Masahiro Shin, Feston Idrizi, Pengpeng Liu, Hassan Gneid, Alireza Edraki, Nathan D Lawson, Jaime A Rivera-Pérez, Erik J Sontheimer, Jonathan K Watts, Craig C Mello
Publikováno v:
eLife, Vol 10 (2021)
Nuclease-directed genome editing is a powerful tool for investigating physiology and has great promise as a therapeutic approach to correct mutations that cause disease. In its most precise form, genome editing can use cellular homology-directed repa
Externí odkaz:
https://doaj.org/article/a4d2a8d974e94e189ee441394236bb61