Evidence map›Paper›PMID 28533404›Full record

ArticleProceedings of the National Academy of Sciences of the United States of America2017

Repression of phosphatidylinositol transfer protein α ameliorates the pathology of Duchenne muscular dystrophy.

Natassia M Vieira, Janelle M Spinazzola, Matthew S Alexander, Yuri B Moreira, Genri Kawahara, Devin E Gibbs, Lillian C Mead, Sergio Verjovski-Almeida, Mayana Zatz, Louis M Kunkel

Registry-linked trialOpen access · bronzeAbstract read
In one paragraph

Article in Proceedings of the National Academy of Sciences of the United States of America, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00390104 (Molecular Analysis of Nucleic Acids Derived From Patients With Neuromuscular Disease and Their Family Members), which is not on this map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
2.2field-weighted citation impact, top 12% 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.

NCT00390104 recruitingnot on this map

Molecular Analysis of Nucleic Acids Derived From Patients With Neuromuscular Disease and Their Family Members

TypeobservationalSponsorBoston Children's HospitalRan2002 to 2027Enrolled1,000ConditionsNeuromuscular, Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
3 · Its place in the literature

Who cites it

26 citing papers in PubMed, 41 citations in OpenAlex.

  1. Article
  2. Muscle-specific increased expression ofProceedings of the National Academy of Sciences of the United States of America · 2025
    Article
  3. Science advances · 2025
    Article
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Courier service for phosphatidylinositol: PITPs deliver on demand.Biochimica et biophysica acta. Molecular and cell biology of lipids · 2021
    Review
  13. Article
  14. Article
  15. PDE10A Inhibition Reduces the Manifestation of Pathology in DMD Zebrafish and Represses the Genetic Modifier PITPNA.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Article
  16. Article
  17. Therapeutic Targeting of PTEN in Duchenne Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Article
  18. Review
  19. Article
  20. 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

10 authors at 3 institutions in 3 countries.

Natassia M VieiraCentro do Genoma Humano e Células-tronco, Instituto de Biociências, Universidade de São Paulo, São Paulo 05508-090, Brazil.
Janelle M SpinazzolaDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115.
Matthew S AlexanderDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115.
Yuri B MoreiraDepartamento de Bioquímica, Instituto de Química, Universidade de São Paulo, São Paulo 05508-900, Brazil.
Genri KawaharaDepartment of Pathophysiology, Tokyo Medical University, Tokyo 160-0022, Japan.
Devin E GibbsDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115.
Lillian C MeadDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115.
Sergio Verjovski-AlmeidaDepartamento de Bioquímica, Instituto de Química, Universidade de São Paulo, São Paulo 05508-900, Brazil.ORCID 0000-0002-6356-2401
Mayana ZatzCentro do Genoma Humano e Células-tronco, Instituto de Biociências, Universidade de São Paulo, São Paulo 05508-090, Brazil.
Louis M KunkelDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA 02115; kunkel@enders.tch.harvard.edu.
Boston Children's Hospital · USUniversidade de São Paulo · BRTokyo Medical University · JP

Funding

SYNAPTIC MECHANISMS IN HIPPOCAMPAL EPILEPTOGENESISP30HD018655 · NICHD · CHILDREN'S HOSPITAL BOSTON · PI POMEROY, SCOTT LOREN · 1985 to 2015
$26.2M
Mouse Neurodevelopmental Behavior CoreU54HD090255 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI POMEROY, SCOTT LOREN · 2016 to 2020
$5.2M
Modulation of Jagged1/Pitpna in DMD as a means of therapyR01AR064300 · NIAMS · BOSTON CHILDREN'S HOSPITAL · PI KUNKEL, LOUIS M · 2014 to 2023
$3.9M
NIAMS NIH HHS R01 AR064300NICHD NIH HHS P30 HD018655NICHD NIH HHS U54 HD090255
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by X-linked inherited mutations in the

Indexed as

AnimalsCell LineDisease Models, AnimalDogsDystrophinHumansMuscle CellsMuscle Fibers, SkeletalMuscle, SkeletalMuscular Dystrophy, AnimalMuscular Dystrophy, DuchenneMutationPhospholipid Transfer ProteinsPhosphorylationProto-Oncogene Proteins c-aktZebrafishDystrophinPhospholipid Transfer ProteinsProto-Oncogene Proteins c-aktDuchenne muscular dystrophygenetic modifierphosphatidylinositol transfer protein-αskeletal muscle

Identifiers

PMID28533404
PMCPMC5468635
OpenAlexW2618941089

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.