Concepedia

Gene Therapy

Gene therapy is a therapeutic biotechnology that modifies genetic material or gene expression in living cells to achieve clinical benefit.

23.1K

Publications

1.5M

Citations

92.8K

Authors

8.2K

Institutions

Publications per year

2017–2026

5.3K

Parents

Children

Publications

23.1K

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

Luigi Naldini, Ulrike Blömer, Philippe Gallay et al. · Science · 1996 · 4.9K citations

, , +14

Silencing of microRNAs in vivo with ‘antagomirs’

Jan Krützfeldt, Nikolaus Rajewsky, Ravi Braich et al. · Nature · 2005 · 4K citations

, , +11

<i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

S. Hacein-Bey-Abina, Christof von Kalle, Manfred Schmidt et al. · Science · 2003 · 3.6K citations

, , +17

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

David Adams, Alejandra González‐Duarte, William O’Riordan et al. · New England Journal of Medicine · 2018 · 2.9K citations · Full text

, , +14

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

Marina Cavazzana, Salima Hacein‐Bey, Geneviève de Saint Basile et al. · Science · 2000 · 2.6K citations

, , +17

1–5 of 23.1K

Authors

92.8K

  1. JM
    James M. Wilson

    University of Pennsylvania, United States

    251 publications · 38.1K citations

    106

    h-index

  2. DT
    David T. Curiel

    University of Alabama at Birmingham, United States

    176 publications · 13K citations

    65

    h-index

  3. WW
    William W. Hauswirth

    University of Florida, United States

    127 publications · 15.2K citations

    64

    h-index

  4. LN
    Luigi Naldini

    The San Raffaele Telethon Institute for Gene Therapy, Italy

    107 publications · 23.2K citations

    61

    h-index

  5. MA
    Mark A. Kay

    Stanford University, United States

    91 publications · 17.2K citations

    61

    h-index

1–5 of 92.8K

Institutions

8.2K

  1. University of Pennsylvania

    Philadelphia, United States

    142 h-index

    3.1K publications

    427.5K citations

  2. Harvard University

    Cambridge, United States

    117 h-index

    2K publications

    218.6K citations

  3. Inserm

    Paris, France

    111 h-index

    4.2K publications

    607.9K citations

  4. University of Florida

    Gainesville, United States

    110 h-index

    2.1K publications

    183.9K citations

  5. National Institutes of Health

    Bethesda, United States

    109 h-index

    2.1K publications

    211.9K citations

  6. Howard Hughes Medical Institute

    Chevy Chase, United States

    107 h-index

    784 publications

    148.8K citations

  7. Stanford University

    Stanford, United States

    101 h-index

    1.4K publications

    171.6K citations

  8. Centre National de la Recherche Scientifique

    Paris, France

    99 h-index

    2K publications

    267K citations

1–8 of 8.2K

Venues

  1. Proceedings of the National Academy of Sciences

    PNAS · 475 publications · 88.2K citations

    161

    h-index

  2. Human Gene Therapy

    1.6K publications · 105.7K citations

    145

    h-index

  3. Molecular Therapy

    1.4K publications · 105.3K citations

    144

    h-index

  4. PubMed

    Repository · 1.4K publications · 68.2K citations

    118

    h-index

  5. Gene Therapy

    1K publications · 63.6K citations

    112

    h-index

1–5

See also

Gene Therapy

Delivery Vectors

Therapeutic Applications

Safety Risks

Regulatory Frameworks