Evidence map›Paper›PMID 38858354›Full record

ArticleNature communications2024

Engineering viral vectors for acoustically targeted gene delivery.

Hongyi R Li, Manwal Harb, John E Heath, James S Trippett, Mikhail G Shapiro, Jerzy O Szablowski

Abstract read
In one paragraph

Article in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing 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

29 citing papers in PubMed.

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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.

Hongyi R LiDivision of Biology and Biological Engineering, California Institute of Technology, Pasadena, CA, USA.
Manwal HarbDepartment of Bioengineering, Rice University, Houston, TX, USA.
John E HeathDivision of Biology and Biological Engineering, California Institute of Technology, Pasadena, CA, USA.ORCID http://orcid.org/0000-0002-5004-2693
James S TrippettDepartment of Bioengineering, Rice University, Houston, TX, USA.
Mikhail G ShapiroDivision of Chemistry and Chemical Engineering, California Institute of Technology, Pasadena, CA, USA. mikhail@caltech.edu.ORCID http://orcid.org/0000-0002-0291-4215
Jerzy O SzablowskiDepartment of Bioengineering, Rice University, Houston, TX, USA. jszab@rice.edu.ORCID http://orcid.org/0000-0001-7851-5408

Funding

Acoustically targeted molecular control of cell type specific neural circuits in non-human primatesUG3MH120102 · NIMH · CALIFORNIA INSTITUTE OF TECHNOLOGY · PI ANDERSEN, RICHARD A, SHAPIRO, MIKHAIL · 2019 to 2021
$3.9M
Brain and Behavior Research Foundation (Brain & Behavior Research Foundation) 22737National Science Foundation (NSF) DGE-2146755NIMH NIH HHS UG3 MH120102U.S. Department of Health & Human Services | NIH | National Institute of Mental Health (NIMH) UG3MH120102
6 · The paper itself

Abstract

Targeted gene delivery to the brain is a critical tool for neuroscience research and has significant potential to treat human disease. However, the site-specific delivery of common gene vectors such as adeno-associated viruses (AAVs) is typically performed via invasive injections, which limit its applicable scope of research and clinical applications. Alternatively, focused ultrasound blood-brain-barrier opening (FUS-BBBO), performed noninvasively, enables the site-specific entry of AAVs into the brain from systemic circulation. However, when used in conjunction with natural AAV serotypes, this approach has limited transduction efficiency and results in substantial undesirable transduction of peripheral organs. Here, we use high throughput in vivo selection to engineer new AAV vectors specifically designed for local neuronal transduction at the site of FUS-BBBO. The resulting vectors substantially enhance ultrasound-targeted gene delivery and neuronal tropism while reducing peripheral transduction, providing a more than ten-fold improvement in targeting specificity in two tested mouse strains. In addition to enhancing the only known approach to noninvasively target gene delivery to specific brain regions, these results establish the ability of AAV vectors to be evolved for specific physical delivery mechanisms.

Indexed as

Blood-Brain BarrierBrainDependovirusGenetic VectorsGene Transfer TechniquesAnimalsFemaleGenetic EngineeringHEK293 CellsHumansMaleMiceMice, Inbred C57BLNeuronsTransduction, Genetic

Identifiers

PMID38858354
PMCPMC11164914

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Registered trials

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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.