Evidence map›Paper›PMID 32668243›Full record

ArticleCell reports2020

In Vivo Repair of a Protein Underlying a Neurological Disorder by Programmable RNA Editing.

John R Sinnamon, Susan Y Kim, Jenna R Fisk, Zhen Song, Hiroyuki Nakai, Sophia Jeng, Shannon K McWeeney, Gail Mandel

Open access · goldAbstract read
In one paragraph

Article in Cell reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed
3.8field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

41 citing papers in PubMed, 61 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Review
  5. Review
  6. Review
  7. Review
  8. Emerging clinical applications of ADAR based RNA editing.Stem cells translational medicine · 2025
    Review
  9. Article
  10. Article
  11. Article
  12. Article
  13. Review
  14. Review
  15. RNA editing: Expanding the potential of RNA therapeutics.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Review
  16. Article
  17. State-of-the-art therapies for Rett syndrome.Developmental medicine and child neurology · 2023
    Review
  18. Review
  19. Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 2 institutions in 1 country.

John R SinnamonVollum Institute, Oregon Health and Science University, Portland, OR 97239, USA.
Susan Y KimVollum Institute, Oregon Health and Science University, Portland, OR 97239, USA.
Jenna R FiskVollum Institute, Oregon Health and Science University, Portland, OR 97239, USA.
Zhen SongDepartment of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239, USA.
Hiroyuki NakaiDepartment of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239, USA.
Sophia JengDivision of Bioinformatics and Computational Biology, Department of Medical Informatics and Clinical Epidemiology, Knight Cancer Institute, Oregon Health and Science University, Portland, OR 97239, USA.
Shannon K McWeeneyDivision of Bioinformatics and Computational Biology, Department of Medical Informatics and Clinical Epidemiology, Knight Cancer Institute, Oregon Health and Science University, Portland, OR 97239, USA.
Gail MandelVollum Institute, Oregon Health and Science University, Portland, OR 97239, USA. Electronic address: mandelg@ohsu.edu.
Oregon Health & Science University · USVollum Institute · US

Funding

Site-directed RNA editing: a new method to correct disease causing mutationsR01NS087726 · NINDS · OREGON HEALTH & SCIENCE UNIVERSITY · PI ADELMAN, JOHN P, BREHM, PAUL · 2013 to 2017
$3.4M
AAV capsid functions, immune evasion and neuronal targeting in mice and NHPR01NS088399 · NINDS · OREGON HEALTH & SCIENCE UNIVERSITY · PI NAKAI, HIROYUKI · 2014 to 2018
$1.7M
Repair of neurological disorders through RNA editingR01NS110868 · NINDS · OREGON HEALTH & SCIENCE UNIVERSITY · PI MANDEL, GAIL · 2020 to 2024
$1.7M
NINDS NIH HHS R01 NS087726NINDS NIH HHS R01 NS088399NINDS NIH HHS R01 NS110868Wellcome Trust
6 · The paper itself

Abstract

Programmable RNA editing is gaining momentum as an approach to repair mutations, but its efficiency in repairing endogenous mutant RNA in complex tissue is unknown. Here we apply this approach to the brain and successfully repair a guanosine-to-adenosine mutation in methyl CpG binding protein 2 RNA that causes the neurodevelopmental disease Rett syndrome. Repair is mediated by hippocampal injections of juvenile Mecp2

Indexed as

Genetic TherapyAmino Acid SequenceAnimalsCell LineGene Expression ProfilingHEK293 CellsHeterochromatinHippocampusHumansMaleMethyl-CpG-Binding Protein 2MiceNervous System DiseasesRNARNA EditingStereotaxic TechniquesHeterochromatinMethyl-CpG-Binding Protein 2RNAADARadenosine deaminase acting on RNAMeCP2Rett syndromeRNA editing

Identifiers

PMID32668243
PMCPMC7449137
OpenAlexW3043502581

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.