Evidence map›Paper›PMID 42388231›Full record

ArticleMaterials today (Kidlington, England)

Biodegradable lipid nanoparticles for genome editing in the brain via intrathecal administration.

Songtao Dong, Lauren Healy, Fanglin Gong, Yue Xu, Yunshu Cai, Nicholas C Solek, Jingan Chen, Muye Zhou, Tyler Thomson, Margarita Savguira and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Materials today (Kidlington, England). The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

15 authors.

Songtao DongLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.
Lauren HealyDepartment of Chemistry, University of Toronto, Toronto, Ontario M5G 1L7, Canada.ORCID 0009-0003-5772-3269
Fanglin GongInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.
Yue XuLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.
Yunshu CaiLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.
Nicholas C SolekInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.ORCID 0009-0008-6336-0229
Jingan ChenInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.
Muye ZhouLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.
Tyler ThomsonInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.ORCID 0000-0002-1434-1813
Margarita SavguiraInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.
Sijin LuozhongLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.
Yanchao ZhangLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.ORCID 0000-0001-7671-725X
Tingzhen HeInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.
Gen LiInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario M5S 3G9, Canada.
Bowen LiLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario M5S 3M2, Canada.ORCID 0000-0001-5006-9143

Funding

Combinatorial design of nonviral base editing systems for treating cystic fibrosis with nonsense mutationsR01HL174773 · NHLBI · UNIVERSITY OF TORONTO · PI Bowen Li · 2024 to 2026
$810k
NHLBI NIH HHS R01 HL174773
6 · The paper itself

Abstract

Messenger RNA (mRNA)-based nonviral delivery of gene editors offers transformative potential for therapeutic genome editing in neurological diseases, but efficient and safe delivery to the brain remains a formidable challenge due to the restrictive blood-brain barrier. Intrathecal administration provides a clinically validated route to bypass this barrier, yet the design principles for biodegradable lipid nanoparticles (LNPs) optimized for central nervous system (CNS) delivery remain poorly defined. Here, we synthesized a 200-member combinatorial library of structurally diverse, biodegradable ionizable lipids using the Passerini three-component reaction. High-throughput

Indexed as

BrainGenome editingIntrathecal administrationIonizable lipidLNPs

Identifiers

PMID42388231
PMCPMC13322347

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.