Evidence map›Paper›PMID 30090842›Full record

ArticleMolecular therapy. Methods & clinical development2018

Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model.

Marie Reine Haddad, Eun-Young Choi, Patricia M Zerfas, Ling Yi, Diego Martinelli, Patricia Sullivan, David S Goldstein, Jose A Centeno, Lauren R Brinster, Martina Ralle and 1 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
2.1field-weighted citation impact, top 14% 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

17 citing papers in PubMed, 26 citations in OpenAlex.

  1. Article
  2. Article
  3. Intravenous AAV9-Science advances · 2025
    Article
  4. Article
  5. Review
  6. Review
  7. Article
  8. Review
  9. Review
  10. Copper metabolism as a unique vulnerability in cancer.Biochimica et biophysica acta. Molecular cell research · 2021
    Review
  11. Article
  12. Article
  13. Article
  14. Article
  15. Review
  16. Copper and the brain noradrenergic system.Journal of biological inorganic chemistry : JBIC : a publication of the Society of Biological Inorganic Chemistry · 2019
    Review
  17. Article
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

11 authors at 5 institutions in 1 country.

Marie Reine HaddadSection on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Eun-Young ChoiSection on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Patricia M ZerfasDiagnostic and Research Services Branch, Office of Research Services, Bethesda, MD, USA.
Ling YiSection on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Diego MartinelliSection on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Patricia SullivanClinical Neurocardiology Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
David S GoldsteinClinical Neurocardiology Section, National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
Jose A CentenoDivision of Biology, Chemistry and Materials Science. Office of Science and Engineering Laboratories (OSEL), US Food and Drug Administration, Silver Spring, MD, USA.
Lauren R BrinsterDiagnostic and Research Services Branch, Office of Research Services, Bethesda, MD, USA.
Martina RalleDepartment of Molecular and Medical Genetics, Oregon Health and Science University, Portland, OR 97239, USA.
Stephen G KalerSection on Translational Neuroscience, Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Eunice Kennedy Shriver National Institute of Child Health and Human Development · USNational Institute of Neurological Disorders and Stroke · USOffice of Research Services · USOregon Health & Science University · USUnited States Food and Drug Administration · US

Funding

Inherited Disorders of Copper TransportZIAHD008768 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI KALER, STEPHEN · 2009 to 2019
$5.5M
Treatment of Catecholaminergic NeurodegenerationZIANS003125 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI GOLDSTEIN, DAVID · 2011 to 2022
$3.2M
The Role of Hepatic Copper Accumulation in Wilson DiseaseR01GM090016 · NIGMS · OREGON HEALTH & SCIENCE UNIVERSITY · PI RALLE, MARTINA · 2010 to 2014
$1.4M
Disorders of Copper TransportZ01HD008768 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI KALER, STEPHEN · 2004 to 2008
$540k
Intramural NIH HHS Z01 HD008768NIGMS NIH HHS R01 GM090016
6 · The paper itself

Abstract

Menkes disease is a lethal neurodegenerative disorder of copper metabolism caused by mutations in an evolutionarily conserved copper transporter, ATP7A. Based on our prior clinical and animal studies, we seek to develop a therapeutic approach suitable for application in affected human subjects, using the

Indexed as

adeno-associated virusATP7Achoroid plexus epitheliacopperdopamine-beta-hydroxylaseMenkes disease

Identifiers

PMID30090842
PMCPMC6080355
OpenAlexW2852732172

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.