Concepedia

Morpholino Oligomers

Morpholino Oligomer is a synthetic nucleic acid analog oligomer in which the ribose phosphate backbone is replaced by a morpholine ring linked through phosphorodiamidate groups, enabling sequence-specific binding to RNA to modulate gene expression without inducing RNase degradation.

261

Publications

16.5K

Citations

1.1K

Authors

309

Institutions

Publications per year

2017–2026

55

Variants

Morpholinos

Parents

Publications

261

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Çirak, Virginia Arechavala‐Gomeza, Michela Guglieri et al. · The Lancet · 2011 · 862 citations · Full text

, , +13

Eteplirsen for the treatment of Duchenne muscular dystrophy

Jerry R. Mendell, Louise R. Rodino‐Klapac, Zarife Sahenk et al. · Annals of Neurology · 2013 · 795 citations · Full text

, , +14

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Maria Kinali, Virginia Arechavala‐Gomeza, Lucy Feng et al. · The Lancet Neurology · 2009 · 639 citations · Full text

, , +17

Longitudinal effect of eteplirsen versus historical control on ambulation in <scp>D</scp> uchenne muscular dystrophy

Jerry R. Mendell, Nathalie Goemans, Linda Lowes et al. · Annals of Neurology · 2015 · 504 citations · Full text

, , +13

Resistance of Morpholino Phosphorodiamidate Oligomers to Enzymatic Degradation

Robert M. Hudziak, Elisabeth Barofsky, Douglas F. Barofsky et al. · Antisense and Nucleic Acid Drug Development · 1996 · 269 citations

, , +16

1–5 of 261

Authors

1.1K

  1. PL
    Patrick L. Iversen

    Sarepta Therapeutics (United States), United States

    67 publications · 5K citations

    47

    h-index

  2. DA
    David A. Stein

    Sarepta Therapeutics (United States), United States

    36 publications · 2.2K citations

    26

    h-index

  3. HM
    Hong M. Moulton

    Oregon State University, United States

    28 publications · 2.6K citations

    23

    h-index

  4. BL
    Bruce L. Geller

    Oregon State University, United States

    16 publications · 883 citations

    16

    h-index

  5. MR
    Mary Rusckowski

    University of Massachusetts Chan Medical School, United States

    17 publications · 489 citations

    14

    h-index

1–5 of 1.1K

Institutions

309

  1. Sarepta Therapeutics (United States)

    Cambridge, United States

    51 h-index

    273 publications

    25.7K citations

  2. Oregon State University

    Corvallis, United States

    24 h-index

    79 publications

    3.2K citations

  3. University of Massachusetts Chan Medical School

    Worcester, United States

    15 h-index

    110 publications

    2.8K citations

  4. The University of Western Australia

    Perth, Australia

    14 h-index

    51 publications

    4.4K citations

  5. Royal Holloway University of London

    Egham, United Kingdom

    12 h-index

    46 publications

    4.6K citations

  6. Corvallis Environmental Center

    Corvallis, United States

    12 h-index

    16 publications

    1.6K citations

  7. Perron Institute for Neurological and Translational Science

    Nedlands, Australia

    11 h-index

    37 publications

    1.2K citations

  8. Murdoch University

    Perth, Australia

    10 h-index

    43 publications

    1.1K citations

1–8 of 309

Venues

  1. Molecular Therapy

    18 publications · 1.3K citations

    17

    h-index

  2. Molecular Therapy — Nucleic Acids

    13 publications · 329 citations

    12

    h-index

  3. Antisense and Nucleic Acid Drug Development

    9 publications · 826 citations

    9

    h-index

  4. PubMed

    Repository · 9 publications · 505 citations

    9

    h-index

  5. Antimicrobial Agents and Chemotherapy

    AAC · 9 publications · 558 citations

    9

    h-index

1–5