Evidence map›Paper›PMID 36712096›Full record

ArticleResearch square2023

Focused ultrasound-mediated brain genome editing.

Kam Leong, Yeh-Hsing Lao, Robin Ji, Joyce Zhou, Kathy Snow, Nancy Kwon, Ethan Saville, Siyu He, Shradha Chauhan, Chun-Wei Chi and 13 more

Abstract readPreprint
In one paragraph

Article in Research square, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

23 authors.

Kam LeongColumbia University.ORCID 0000-0002-8133-4955
Yeh-Hsing LaoUniversity at Buffalo.ORCID 0000-0002-7990-199X
Robin JiColumbia University.
Joyce ZhouColumbia University.
Kathy SnowThe Jackson Laboratory.
Nancy KwonColumbia University.
Ethan SavilleThe Jackson Laboratory.
Siyu HeColumbia University.
Shradha ChauhanColumbia University.
Chun-Wei ChiUniversity at Buffalo.
Malika DattaColumbia University.
Hairong ZhangColumbia University.
Chai Hoon QuekColumbia University.
Sarah CaiColumbia University.
Mingqiang LiSun Yat-sen University.
Yaned GaitanThe Jackson Laboratory.
Lawrence BechtelThe Jackson Laboratory.
Shih-Ying WuColumbia University.
Cathleen LutzJackson Laboratory.ORCID 0000-0003-2502-1900
Raju TomerColumbia University.ORCID 0000-0002-4229-2860
Stephen MurrayThe Jackson Laboratory, 600 Main Street, Bar Harbor, Maine 04609.ORCID 0000-0002-0594-1702
Alejandro ChavezUniversity of California San Diego.
Elisa KonofagouColumbia University.ORCID 0000-0002-9636-7936

Funding

Tumor Biology and Microenvironment ProgramP30CA013696 · NCI · COLUMBIA UNIV NEW YORK MORNINGSIDE · PI Anil K Rustgi · 1985 to 2026
$115.3M
The Jackson Laboratory Gene Editing Testing Center (JAX-GETC)U42OD026635 · OD · JACKSON LABORATORY · PI MURRAY, STEPHEN A · 2018 to 2022
$4.6M
A comparative approach for decomposing the mammalian brain architectural complexityDP2MH119423 · NIMH · COLUMBIA UNIV NEW YORK MORNINGSIDE · PI TOMER, RAJU · 2018 to 2018
$2.4M
Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative DisordersUG3NS115598 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI LEONG, KAM W · 2019 to 2021
$2.3M
NCI NIH HHS P30 CA013696NIH HHS U42 OD026635NIMH NIH HHS DP2 MH119423NINDS NIH HHS UG3 NS115598
6 · The paper itself

Abstract

Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions of the brain. We demonstrate a safe and effective gene editing technique by using focused ultrasound (FUS) to transiently open the BBB for the transport of intravenously delivered CRISPR/Cas9 machinery to the brain.

Identifiers

PMID36712096
PMCPMC9882596

What OpenQuestion holds

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LicenceCC BY
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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.