Evidence map›Paper›PMID 31534984›Full record

ArticleMolecular therapy. Methods & clinical development2019

Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1.

Shilpa Prabhakar, Pike See Cheah, Xuan Zhang, Max Zinter, Maria Gianatasio, Eloise Hudry, Roderick T Bronson, David J Kwiatkowski, Anat Stemmer-Rachamimov, Casey A Maguire and 3 more

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

Article in Molecular therapy. Methods & clinical development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
1.7field-weighted citation impact, top 16% 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

19 citing papers in PubMed, 52 citations in OpenAlex.

  1. [Adeno-associated virus-mediated gene therapy for genetic epilepsy: prospects and challenges].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026
    Review
  2. Tuberous sclerosis complex.Nature reviews. Disease primers · 2026
    Review
  3. Article
  4. Review
  5. Review
  6. Article
  7. Review
  8. Article
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  10. Article
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  12. Review
  13. Article
  14. Review
  15. Clinical Trial Design for Disease-Modifying Therapies for Genetic Epilepsies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021
    Review
  16. Review
  17. Review
  18. Article
  19. TSC1 Gene Therapy AAVerts Neurological Associated Mortality.Molecular therapy : the journal of the American Society of Gene Therapy · 2019
    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

13 authors at 5 institutions in 2 countries.

Shilpa PrabhakarMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Pike See CheahMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Xuan ZhangMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Max ZinterMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Maria GianatasioDepartment of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Eloise HudryMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Roderick T BronsonRodent Histopathology Core Facility, Harvard Medical School, Boston, MA, USA.
David J KwiatkowskiBrigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Anat Stemmer-RachamimovDepartment of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Casey A MaguireMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Miguel Sena-EstevesDepartment of Neurology, Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Bakhos A TannousMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Xandra O BreakefieldMolecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Neurodiscovery Center, Harvard Medical School, Charlestown, MA, USA.
Harvard University · USMassachusetts General Hospital · USHarvard NeuroDiscovery Center · USBrigham and Women's Hospital · USUniversity of Massachusetts Chan Medical School · US

Funding

Vector Query and Therapy For TSC and NF2 LesionsP01NS024279 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS · 1987 to 2012
$14.7M
Interdepartmental Neuroscience CenterP30NS045776 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS, TANNOUS, BAKHOS A · 2003 to 2018
$11.1M
NINDS NIH HHS P01 NS024279NINDS NIH HHS P30 NS045776
6 · The paper itself

Abstract

Tuberous sclerosis complex (TSC) is a tumor suppressor syndrome caused by mutations in

Indexed as

braingene therapyneurologytumor suppressor

Identifiers

PMID31534984
PMCPMC6745533
OpenAlexW2967739194

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.