Evidence map›Paper›PMID 37485250›Full record

ReviewMed-X2023

Non-viral approaches for gene therapy and therapeutic genome editing across the blood-brain barrier.

Ruosen Xie, Yuyuan Wang, Jacobus C Burger, Dongdong Li, Min Zhu, Shaoqin Gong

Open access · hybridAbstract readReview
In one paragraph

Review in Med-X, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
2.6field-weighted citation impact, top 10% 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

13 citing papers in PubMed, 17 citations in OpenAlex.

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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 at 2 institutions in 1 country.

Ruosen XieDepartment of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705 USA.
Yuyuan WangDepartment of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705 USA.
Jacobus C BurgerWisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715 USA.
Dongdong LiDepartment of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705 USA.
Min ZhuWisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715 USA.
Shaoqin GongDepartment of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705 USA.
University of Wisconsin–Madison · USWisconsin Institutes for Discovery · US

Funding

Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the BrainUH3NS111688 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI EMBORG, MARINA, GONG, SHAOQIN - · 2021 to 2022
$2.4M
NINDS NIH HHS UH3 NS111688
6 · The paper itself

Abstract

The success of brain-targeted gene therapy and therapeutic genome editing hinges on the efficient delivery of biologics bypassing the blood-brain barrier (BBB), which presents a significant challenge in the development of treatments for central nervous system disorders. This is particularly the case for nucleic acids and genome editors that are naturally excluded by the BBB and have poor chemical stability in the bloodstream and poor cellular uptake capability, thereby requiring judiciously designed nanovectors administered systemically for intracellular delivery to brain cells such as neurons. To overcome this obstacle, various strategies for bypassing the BBB have been developed in recent years to deliver biologics to the brain via intravenous administration using non-viral vectors. This review summarizes various brain targeting strategies and recent representative reports on brain-targeted non-viral delivery systems that allow gene therapy and therapeutic genome editing via intravenous administration, and highlights ongoing challenges and future perspectives for systemic delivery of biologics to the brain via non-viral vectors.

Indexed as

Blood–brain barrierBrain-targeting strategiesGene therapyGenome editingNon-viral vectors

Identifiers

PMID37485250
PMCPMC10357415
OpenAlexW4383743486

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.