Literature DB >> 35613622

Base editing the synapse: Modeling a complex neurological disorder in non-human primates.

Benjamin L Prosser1, Ingo Helbig2.   

Abstract

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Year:  2022        PMID: 35613622      PMCID: PMC9171259          DOI: 10.1016/j.ymthe.2022.05.009

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   12.910


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  9 in total

Review 1.  STXBP1 encephalopathy: A neurodevelopmental disorder including epilepsy.

Authors:  Hannah Stamberger; Marina Nikanorova; Marjolein H Willemsen; Patrizia Accorsi; Marco Angriman; Hartmut Baier; Ira Benkel-Herrenbrueck; Valérie Benoit; Mauro Budetta; Almuth Caliebe; Gaetano Cantalupo; Giuseppe Capovilla; Gianluca Casara; Carolina Courage; Marie Deprez; Anne Destrée; Robertino Dilena; Corrie E Erasmus; Madeleine Fannemel; Roar Fjær; Lucio Giordano; Katherine L Helbig; Henrike O Heyne; Joerg Klepper; Gerhard J Kluger; Damien Lederer; Monica Lodi; Oliver Maier; Andreas Merkenschlager; Nina Michelberger; Carlo Minetti; Hiltrud Muhle; Judith Phalin; Keri Ramsey; Antonino Romeo; Jens Schallner; Ina Schanze; Marwan Shinawi; Kristel Sleegers; Katalin Sterbova; Steffen Syrbe; Monica Traverso; Andreas Tzschach; Peter Uldall; Rudy Van Coster; Helene Verhelst; Maurizio Viri; Susan Winter; Markus Wolff; Martin Zenker; Leonardo Zoccante; Peter De Jonghe; Ingo Helbig; Pasquale Striano; Johannes R Lemke; Rikke S Møller; Sarah Weckhuysen
Journal:  Neurology       Date:  2016-02-10       Impact factor: 9.910

2.  Base-edited cynomolgus monkeys mimic core symptoms of STXBP1 encephalopathy.

Authors:  Zongyang Lu; Siting He; Jian Jiang; Ling Zhuang; Yan Wang; Guang Yang; Xiaoyu Jiang; Yanhong Nie; Jiqiang Fu; Xiaotong Zhang; Yong Lu; Xinyan Bian; Hung-Chun Chang; Zhiqi Xiong; Xingxu Huang; Zhen Liu; Qiang Sun
Journal:  Mol Ther       Date:  2022-03-11       Impact factor: 12.910

3.  In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.

Authors:  Kiran Musunuru; Alexandra C Chadwick; Taiji Mizoguchi; Sara P Garcia; Jamie E DeNizio; Caroline W Reiss; Kui Wang; Sowmya Iyer; Chaitali Dutta; Victoria Clendaniel; Michael Amaonye; Aaron Beach; Kathleen Berth; Souvik Biswas; Maurine C Braun; Huei-Mei Chen; Thomas V Colace; John D Ganey; Soumyashree A Gangopadhyay; Ryan Garrity; Lisa N Kasiewicz; Jennifer Lavoie; James A Madsen; Yuri Matsumoto; Anne Marie Mazzola; Yusuf S Nasrullah; Joseph Nneji; Huilan Ren; Athul Sanjeev; Madeleine Shay; Mary R Stahley; Steven H Y Fan; Ying K Tam; Nicole M Gaudelli; Giuseppe Ciaramella; Leslie E Stolz; Padma Malyala; Christopher J Cheng; Kallanthottathil G Rajeev; Ellen Rohde; Andrew M Bellinger; Sekar Kathiresan
Journal:  Nature       Date:  2021-05-19       Impact factor: 69.504

4.  Protein instability, haploinsufficiency, and cortical hyper-excitability underlie STXBP1 encephalopathy.

Authors:  Jovana Kovacevic; Gregoire Maroteaux; Desiree Schut; Maarten Loos; Mohit Dubey; Julika Pitsch; Esther Remmelink; Bastijn Koopmans; James Crowley; L Niels Cornelisse; Patrick F Sullivan; Susanne Schoch; Ruud F Toonen; Oliver Stiedl; Matthijs Verhage
Journal:  Brain       Date:  2018-05-01       Impact factor: 13.501

5.  Mechanism-based rescue of Munc18-1 dysfunction in varied encephalopathies by chemical chaperones.

Authors:  Noah Guy Lewis Guiberson; André Pineda; Debra Abramov; Parinati Kharel; Kathryn E Carnazza; Rachel T Wragg; Jeremy S Dittman; Jacqueline Burré
Journal:  Nat Commun       Date:  2018-09-28       Impact factor: 14.919

6.  Stxbp1/Munc18-1 haploinsufficiency impairs inhibition and mediates key neurological features of STXBP1 encephalopathy.

Authors:  Wu Chen; Zhao-Lin Cai; Eugene S Chao; Hongmei Chen; Colleen M Longley; Shuang Hao; Hsiao-Tuan Chao; Joo Hyun Kim; Jessica E Messier; Huda Y Zoghbi; Jianrong Tang; John W Swann; Mingshan Xue
Journal:  Elife       Date:  2020-02-19       Impact factor: 8.140

Review 7.  Moving toward genome-editing therapies for cardiovascular diseases.

Authors:  Kiran Musunuru
Journal:  J Clin Invest       Date:  2022-01-04       Impact factor: 14.808

8.  In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.

Authors:  Tanja Rothgangl; Melissa K Dennis; Paulo J C Lin; Rurika Oka; Dominik Witzigmann; Lukas Villiger; Weihong Qi; Martina Hruzova; Lucas Kissling; Daniela Lenggenhager; Costanza Borrelli; Sabina Egli; Nina Frey; Noëlle Bakker; John A Walker; Anastasia P Kadina; Denis V Victorov; Martin Pacesa; Susanne Kreutzer; Zacharias Kontarakis; Andreas Moor; Martin Jinek; Drew Weissman; Markus Stoffel; Ruben van Boxtel; Kevin Holden; Norbert Pardi; Beat Thöny; Johannes Häberle; Ying K Tam; Sean C Semple; Gerald Schwank
Journal:  Nat Biotechnol       Date:  2021-05-19       Impact factor: 54.908

9.  Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.

Authors:  Alexis C Komor; Yongjoo B Kim; Michael S Packer; John A Zuris; David R Liu
Journal:  Nature       Date:  2016-04-20       Impact factor: 49.962

  9 in total

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