Evidence map›Paper›PMID 27933308›Full record

ArticleMolecular therapy. Methods & clinical development2016

Tailored transgene expression to specific cell types in the central nervous system after peripheral injection with AAV9.

Jonathan Dashkoff, Eli P Lerner, Nhi Truong, Jacob A Klickstein, Zhanyun Fan, Dakai Mu, Casey A Maguire, Bradley T Hyman, Eloise Hudry

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed
8.1field-weighted citation impact, top 2% 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

30 citing papers in PubMed, 52 citations in OpenAlex.

  1. Systemic dual-gene therapy reverses biochemical intoxication in the central metabolic compartment of Bckdha-/- mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  2. Review
  3. Article
  4. Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9.International journal of molecular sciences · 2023
    Article
  5. Review
  6. Review
  7. Review
  8. Article
  9. Article
  10. Review
  11. Article
  12. Review
  13. Article
  14. Selection of an Efficient AAV Vector for Robust CNS Transgene Expression.Molecular therapy. Methods & clinical development · 2019
    Article
  15. Article
  16. Article
  17. Article
  18. Article
  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

9 authors at 3 institutions in 1 country.

Jonathan DashkoffDepartment of Anatomy and Neurobiology, Boston University School of Medicine, Boston, Massachusetts, USA; MassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School, Charlestown, Massachusetts, USA.
Eli P LernerMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Nhi TruongMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Jacob A KlicksteinMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Zhanyun FanMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Dakai MuDepartment of Neurology, The Massachusetts General Hospital, and NeuroDiscovery Center, Harvard Medical School , Boston, Massachusetts, USA.
Casey A MaguireDepartment of Neurology, The Massachusetts General Hospital, and NeuroDiscovery Center, Harvard Medical School , Boston, Massachusetts, USA.
Bradley T HymanMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Eloise HudryMassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital and Harvard Medical School , Charlestown, Massachusetts, USA.
Harvard University · USMassachusetts General Hospital · USMaineGeneral Medical Center · US

Funding

Interdepartmental Neuroscience CenterP30NS045776 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS, TANNOUS, BAKHOS A · 2003 to 2018
$11.1M
Novel Strategies and Mechanisms to Target APOE and Alzheimer's DiseaseRF1AG047644 · NIA · WASHINGTON UNIVERSITY · PI HOLTZMAN, DAVID M., HYMAN, BRADLEY T. · 2019 to 2019
$3.8M
Role of apolipoprotein E in age and amyloid beta related neuronal dysfunctionR00AG047336 · NIA · MASSACHUSETTS GENERAL HOSPITAL · PI HUDRY, ELOISE · 2017 to 2019
$747k
A hybrid microvesicle/virus vector for targeted gene transfer to the brainR21NS081374 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI MAGUIRE, CASEY A · 2012 to 2013
$449k
Role of apolipoprotein E in age and amyloid beta related neuronal dysfunctionK99AG047336 · NIA · MASSACHUSETTS GENERAL HOSPITAL · PI HUDRY, ELOISE · 2015 to 2016
$273k
NIA NIH HHS K99 AG047336NIA NIH HHS R00 AG047336NIA NIH HHS RF1 AG047644NINDS NIH HHS P30 NS045776NINDS NIH HHS R21 NS081374
6 · The paper itself

Abstract

The capacity of certain adeno-associated virus (AAV) vectors to cross the blood-brain barrier after intravenous delivery offers a unique opportunity for noninvasive brain delivery. However, without a well-tailored system, the use of a peripheral route injection may lead to undesirable transgene expression in nontarget cells or organs. To refine this approach, the present study characterizes the transduction profiles of new self-complementary AAV9 (scAAV9) expressing the green fluorescent protein (GFP) either under an astrocyte (glial fibrillary acidic (GFA) protein) or neuronal (Synapsin (Syn)) promoter, after intravenous injection of adult mice (2 × 10

Identifiers

PMID27933308
PMCPMC5142512
OpenAlexW2559866176

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.