Concepedia

Disease-causing Mutation Correction

Disease-Causing Mutation Correction is a targeted genome editing strategy that belongs to the class of molecular therapeutic interventions, specifically distinguished by its precise modification of pathogenic genetic variants using engineered nucleases such as Zinc Finger Nucleases to restore normal gene function.

298

Publications

26.5K

Citations

2.4K

Authors

627

Institutions

Publications per year

2017–2026

176

Parents

Publications

298

Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing

Zhen Liu, Zongyang Lu, Guang Yang et al. · Nature Communications · 2018 · 2.1K citations · Full text

, , +16

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Christopher E. Nelson, Chady H. Hakim, David G. Ousterout et al. · Science · 2015 · 1.1K citations

, , +17

Correction of a pathogenic gene mutation in human embryos

Hong Ma, Nuria Martí‐Gutiérrez, Sang-Wook Park et al. · Nature · 2017 · 951 citations

, , +12

Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA

Chengzu Long, John McAnally, John M. Shelton et al. · Science · 2014 · 721 citations · Full text

, , +17

Correction of a Genetic Defect by Nuclear Transplantation and Combined Cell and Gene Therapy

William M. Rideout, Konrad Hochedlinger, Michael Kyba et al. · Cell · 2002 · 598 citations · Full text

, , +16

1–5 of 298

Authors

2.4K

  1. ZR
    Zulema Romero

    University of California, Los Angeles, United States

    6 publications · 458 citations

    6

    h-index

  2. DB
    Donald B. Kohn

    Children's Hospital of Los Angeles, United States

    6 publications · 458 citations

    6

    h-index

  3. EN
    Eric N. Olson

    The University of Texas Southwestern Medical Center, United States

    6 publications · 1.9K citations

    6

    h-index

  4. RB
    Rhonda Bassel‐Duby

    The University of Texas Southwestern Medical Center, United States

    6 publications · 1.9K citations

    6

    h-index

  5. BC
    Beatriz Campo-Fernández

    University of California, Los Angeles, United States

    6 publications · 458 citations

    6

    h-index

1–5 of 2.4K

Institutions

627

  1. Harvard University

    Cambridge, United States

    18 h-index

    64 publications

    9K citations

  2. Inserm

    Paris, France

    11 h-index

    42 publications

    2.9K citations

  3. The University of Texas Southwestern Medical Center

    Dallas, United States

    10 h-index

    61 publications

    16.1K citations

  4. Children's Hospital of Philadelphia

    Philadelphia, United States

    10 h-index

    49 publications

    6.8K citations

  5. Massachusetts General Hospital

    Boston, United States

    10 h-index

    38 publications

    6K citations

  6. Stanford University

    Stanford, United States

    9 h-index

    78 publications

    12.2K citations

  7. Broad Institute

    Cambridge, United States

    9 h-index

    22 publications

    6.2K citations

  8. Howard Hughes Medical Institute

    Chevy Chase, United States

    9 h-index

    25 publications

    4.6K citations

1–8 of 627

Venues

  1. Molecular Therapy

    23 publications · 2K citations

    22

    h-index

  2. Nature Communications

    17 publications · 3.6K citations

    17

    h-index

  3. Molecular Therapy — Nucleic Acids

    13 publications · 584 citations

    12

    h-index

  4. Science

    8 publications · 3.3K citations

    8

    h-index

  5. Nucleic Acids Research

    7 publications · 449 citations

    7

    h-index

1–5