Evidence map›Paper›PMID 26905292›Full record

ReviewNeuropharmacology2017

Viral vectors for therapy of neurologic diseases.

Sourav R Choudhury, Eloise Hudry, Casey A Maguire, Miguel Sena-Esteves, Xandra O Breakefield, Paola Grandi

Abstract readReview
In one paragraph

Review in Neuropharmacology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 105 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
105citing papers in PubMed, 1 pooled it
18.3field-weighted citation impact, top 1% 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

105 citing papers in PubMed, 1 synthesis or guideline pooled it, 182 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
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  5. Review
  6. Review
  7. Review
  8. Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  9. Review
  10. Article
  11. Review
  12. Review
  13. Review
  14. Article
  15. Review
  16. State-of-the-art gene therapy in epilepsy.Current opinion in neurology · 2025
    Review
  17. Article
  18. Review
  19. Review
  20. Riboflavin transporter deficiency:Frontiers in cellular neuroscience · 2025
    Article

45 more citing papers are in PubMed but not listed here.

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

6 authors at 5 institutions in 1 country.

Sourav R ChoudhuryDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA 01605, USA; Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address: sourav.rc@gmail.com.
Eloise HudryAlzheimer's Disease Research Unit, Harvard Medical School & Massachusetts General Hospital, Charlestown, MA 02129, USA. Electronic address: ehudry@mgh.harvard.edu.
Casey A MaguireDepartment of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital and NeuroDiscovery Center, Harvard Medical School, Boston, MA 02114, USA. Electronic address: cmaguire@mgh.harvard.edu.
Miguel Sena-EstevesDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA 01605, USA; Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address: miguel.esteves@umassmed.edu.
Xandra O BreakefieldDepartment of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital and NeuroDiscovery Center, Harvard Medical School, Boston, MA 02114, USA. Electronic address: breakefield@hms.harvard.edu.
Paola GrandiDepartment of Neurological Surgery, University of Pittsburgh Cancer Institute, Pittsburgh, PA 15219, USA. Electronic address: pag24@pitt.edu.
University of Massachusetts Chan Medical School · USHarvard NeuroDiscovery Center · USHarvard University · USMassachusetts General Hospital · USNeurological Surgery · US

Funding

The role of microvesicles in giloma virotherapyP01CA069246 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI CHIOCCA, E. ANTONIO · 1996 to 2021
$30.9M
The Tumor Microenvironmental Barrier to Effective Viral OncolysisP01CA163205 · NCI · OHIO STATE UNIVERSITY · PI KEITH LLOYD LIGON · 2013 to 2026
$26.0M
Transfer of microRNA regulators from glioblastoma to brain microenvironment U19CA179563 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS, CHAREST, ALAIN · 2013 to 2017
$8.6M
Gene Therapy for Neurodegenerative Lysosomal Storage DiseasesR01HD060576 · NICHD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ESTEVES, MIGUEL S, MARTIN, DOUGLAS R · 2010 to 2013
$2.0M
Development of CNS-targeted AAV vectorsR01NS066310 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ESTEVES, MIGUEL S · 2009 to 2012
$1.3M
NCI NIH HHS P01 CA069246NCI NIH HHS P01 CA163205NCI NIH HHS U19 CA179563NICHD NIH HHS R01 HD060576NINDS NIH HHS R01 NS066310
6 · The paper itself

Abstract

Neurological disorders - disorders of the brain, spine and associated nerves - are a leading contributor to global disease burden with a shockingly large associated economic cost. Various treatment approaches - pharmaceutical medication, device-based therapy, physiotherapy, surgical intervention, among others - have been explored to alleviate the resulting extent of human suffering. In recent years, gene therapy using viral vectors - encoding a therapeutic gene or inhibitory RNA into a "gutted" viral capsid and supplying it to the nervous system - has emerged as a clinically viable option for therapy of brain disorders. In this Review, we provide an overview of the current state and advances in the field of viral vector-mediated gene therapy for neurological disorders. Vector tools and delivery methods have evolved considerably over recent years, with the goal of providing greater and safer genetic access to the central nervous system. Better etiological understanding of brain disorders has concurrently led to identification of improved therapeutic targets. We focus on the vector technology, as well as preclinical and clinical progress made thus far for brain cancer and various neurodegenerative and neurometabolic disorders, and point out the challenges and limitations that accompany this new medical modality. Finally, we explore the directions that neurological gene therapy is likely to evolve towards in the future. This article is part of the Special Issue entitled "Beyond small molecules for neurological disorders".

Indexed as

AnimalsBrain NeoplasmsGenetic TherapyGenetic VectorsGene Transfer TechniquesHumansNervous System DiseasesBrain tumorsGene therapyLysosomal storage diseasesMetabolic diseasesNeurodegenerationViral vectors

Identifiers

PMID26905292
PMCPMC5929167
OpenAlexW2280490150

What OpenQuestion holds

Textmetadata
LicenceTDM
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.