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Akademický článek

Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope

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

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Elektronická kniha

Nucleic Acids in Chemistry and Biology

Autor: G Michael Blackburn, Martin Egli, Michael J Gait, Jonathan K Watts

The structure, function and reactions of nucleic acids are central to molecular biology and medicine and are crucial for understanding of the ever-expanding range of complex biological processes involved which are central to life. Revised, extended,

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Akademický článek

EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

Autor: Minggang Fang, Sara K. Deibler, Pranathi Meda Krishnamurthy, Feng Wang, Paola Rodriguez, Shahid Banday, Ching-Man Virbasius, Miguel Sena-Esteves, Jonathan K. Watts, Michael R. Green

Publikováno v: Frontiers in Neuroscience, Vol 18 (2024)

Fragile X Syndrome (FXS) is a neurological disorder caused by epigenetic silencing of the FMR1 gene. Reactivation of FMR1 is a potential therapeutic approach for FXS that would correct the root cause of the disease. Here, using a candidate-based shRN

Externí odkaz: https://doaj.org/article/3a274fd2567945d28c750f6d92ecc94f

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Akademický článek

Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity

Autor: Hassan H. Fakih, Qi Tang, Ashley Summers, Minwook Shin, Julianna E. Buchwald, Rosemary Gagnon, Vignesh N. Hariharan, Dimas Echeverria, David A. Cooper, Jonathan K. Watts, Anastasia Khvorova, Hanadi F. Sleiman

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 34, Iss , Pp 102080- (2023)

Although an increasing number of small interfering RNA (siRNA) therapies are reaching the market, the challenge of efficient extra-hepatic delivery continues to limit their full therapeutic potential. Drug delivery vehicles and hydrophobic conjugates

Externí odkaz: https://doaj.org/article/b2737ae9f41849d78fd6f4851c674ed3

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Akademický článek

Structure of the catalytically active APOBEC3G bound to a DNA oligonucleotide inhibitor reveals tetrahedral geometry of the transition state

Autor: Atanu Maiti, Adam K. Hedger, Wazo Myint, Vanivilasini Balachandran, Jonathan K. Watts, Celia A. Schiffer, Hiroshi Matsuo

Publikováno v: Nature Communications, Vol 13, Iss 1, Pp 1-10 (2022)

Here, the enzymatic activity of APOBEC3G resulting in conversion of 2′-deoxy-zebularine into a hydration product allowed the authors to capture the transition state, which provides a blueprint for designing a new class of transition state-mimicking

Externí odkaz: https://doaj.org/article/9b59b40269cb4ef4b61dc593fa6d83f7

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Akademický článek

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

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

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