Evidence map›Paper›PMID 29596011›Full record

ArticleHuman gene therapy methods2018

Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space.

Dan Wang, Jia Li, Karen Tran, Daniel R Burt, Li Zhong, Guangping Gao

Abstract read
In one paragraph

Article in Human gene therapy methods, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
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

6 citing papers in PubMed.

  1. Current trends in gene therapy to treat inherited disorders of the brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  2. Article
  3. Article
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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.

Dan Wang1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Jia Li1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Karen Tran1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Daniel R Burt1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Li Zhong1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Guangping Gao1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.

Funding

Viral escape from AAV expressed transgenesP01AI100263 · NIAID · SCRIPPS FLORIDA · PI FARZAN, MICHAEL R. · 2012 to 2016
$11.9M
Viral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI FLOTTE, TERENCE R. · 2016 to 2020
$11.3M
Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7M
Next Generation of Recombinant AAV Serotype Vectors for Gene TherapyR01HL097088 · NHLBI · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, HERZOG, ROLAND W. · 2010 to 2018
$5.4M
NHLBI NIH HHS P01 HL131471NHLBI NIH HHS R01 HL097088NIAID NIH HHS P01 AI100263NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

Recombinant adeno-associated viruses (rAAVs) are the leading in vivo gene delivery platform, and have been extensively studied in gene therapy targeting various tissues, including the central nervous system (CNS). A single-bolus rAAV injection to the cerebrospinal fluid (CSF) space has been widely used to target the CNS, but it suffers from several drawbacks, such as leakage to peripheral tissues. Here, a protocol is described using an osmotic pump to infuse rAAV slowly into the mouse CSF space. Compared to the single-bolus injection technique, pump infusion can lead to higher CNS transduction and lower transduction in the peripheral tissues.

Indexed as

AnimalsBrainCatheters, IndwellingCerebrospinal FluidDependovirusFemaleGenetic TherapyGenetic VectorsHumansInfusion Pumps, ImplantableInfusions, IntraventricularInfusions, SpinalMaleMiceMice, Inbred C57BLOsmotic Pressureadeno-associated viruscentral nervous systemgene therapyintrathecal delivery

Identifiers

PMID29596011
PMCPMC5910051

What OpenQuestion holds

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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.