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Genetics, Gene Therapy & Genetic Disease

  • Open Access
    MET mutation causes muscular dysplasia and arthrogryposis
    <em>MET</em> mutation causes muscular dysplasia and arthrogryposis
    Hang Zhou, Chengjie Lian, Tingting Wang, Xiaoming Yang, Caixia Xu, Deying Su, Shuhui Zheng, Xiangyu Huang, Zhiheng Liao, Taifeng Zhou, Xianjian Qiu, Yuyu Chen, Bo Gao, Yongyong Li, Xudong Wang, Guoling You, Qihua Fu, Christina Gurnett, Dongsheng Huang, Peiqiang Su
    Published online 18.02.2019
    • Genetics, Gene Therapy & Genetic Disease
    • Musculoskeletal System
  • Open Access
    Innovation and competition in advanced therapy medicinal products
    Innovation and competition in advanced therapy medicinal products
    Enrique Seoane‐Vazquez, Vaishali Shukla, Rosa Rodriguez‐Monguio
    Published online 15.02.2019
    • Genetics, Gene Therapy & Genetic Disease
    • Stem Cells
  • Open Access
    Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives
    Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives
    Luigi Naldini
    Published online 22.01.2019
    • Genetics, Gene Therapy & Genetic Disease
    • Haematology
    • Stem Cells
  • Open Access
    Repeat‐associated non‐AUG translation in C9orf72‐ALS/FTD is driven by neuronal excitation and stress
    Repeat‐associated non‐AUG translation in C9orf72‐ALS/FTD is driven by neuronal excitation and stress
    Thomas Westergard, Kevin McAvoy, Katelyn Russell, Xinmei Wen, Yu Pang, Brandie Morris, Piera Pasinelli, Davide Trotti, Aaron Haeusler
    Published online 07.01.2019
    • Genetics, Gene Therapy & Genetic Disease
    • Neuroscience
  • Open Access
    Split otoferlins reunited
    Split otoferlins reunited
    Jeffrey R Holt, Gwenaelle SG Geleoc
    Published online 20.12.2018
    • Genetics, Gene Therapy & Genetic Disease
    • Neuroscience
  • Open Access
    How much does a disrupted mitochondrial network influence neuronal dysfunction?
    How much does a disrupted mitochondrial network influence neuronal dysfunction?
    Zofia MA Chrzanowska‐Lightowlers, Robert N Lightowlers
    Published online 14.12.2018
    • Genetics, Gene Therapy & Genetic Disease
    • Neuroscience
  • Open Access
    APOPT1/COA8 assists COX assembly and is oppositely regulated by UPS and ROS
    APOPT1/COA8 assists COX assembly and is oppositely regulated by UPS and ROS
    Alba Signes, Raffaele Cerutti, Anna S Dickson, Cristiane Benincá, Elizabeth C Hinchy, Daniele Ghezzi, Rosalba Carrozzo, Enrico Bertini, Michael P Murphy, James A Nathan, Carlo Viscomi, Erika Fernandez‐Vizarra, Massimo Zeviani
    Published online 14.12.2018
    • Genetics, Gene Therapy & Genetic Disease
  • Open Access
    Alternative oxidase‐mediated respiration prevents lethal mitochondrial cardiomyopathy
    Alternative oxidase‐mediated respiration prevents lethal mitochondrial cardiomyopathy
    Jayasimman Rajendran, Janne Purhonen, Saara Tegelberg, Olli‐Pekka Smolander, Matthias Mörgelin, Jan Rozman, Valerie Gailus‐Durner, Helmut Fuchs, Martin Hrabe de Angelis, Petri Auvinen, Eero Mervaala, Howard T Jacobs, Marten Szibor, Vineta Fellman, Jukka Kallijärvi
    Published online 10.12.2018
    • Cardiovascular System
    • Genetics, Gene Therapy & Genetic Disease
  • Open Access
    Sea squirt alternative oxidase bypasses fatal mitochondrial heart disease
    Sea squirt alternative oxidase bypasses fatal mitochondrial heart disease
    Ann Saada
    Published online 10.12.2018
    • Cardiovascular System
    • Genetics, Gene Therapy & Genetic Disease
  • Open Access
    Murine MPDZ‐linked hydrocephalus is caused by hyperpermeability of the choroid plexus
    Murine <em>MPDZ</em>‐linked hydrocephalus is caused by hyperpermeability of the choroid plexus
    Junning Yang, Claire Simonneau, Robert Kilker, Laura Oakley, Matthew D Byrne, Zuzana Nichtova, Ioana Stefanescu, Fnu Pardeep‐Kumar, Sushil Tripathi, Eric Londin, Pascale Saugier‐Veber, Belinda Willard, Mathew Thakur, Stephen Pickup, Hiroshi Ishikawa, Horst Schroten, Richard Smeyne, Arie Horowitz
    Published online 05.12.2018
    • Genetics, Gene Therapy & Genetic Disease
    • Neuroscience

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