Evidence map›Paper›PMID 42597608›Full record

ArticleMolecular therapy. Nucleic acids2026

Design and development of lipid nanoparticle formulations for brain gene therapy.

Sarah B Thomson, Alissandra de Moura Gomes, Pardis Kazemian, Daniel Z Kurek, Jayesh A Kulkarni, Fariba Sadaati, Pamela K Wagner, Terri L Petkau, Marco A Ciufolini, Pieter R Cullis and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. 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

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

11 authors.

Sarah B ThomsonUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.
Alissandra de Moura GomesUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.
Pardis KazemianUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.
Daniel Z KurekNanoVation Therapeutics, 2665 East Mall, 2 Floor, Vancouver, BC V6T 1Z4, Canada.
Jayesh A KulkarniNanoVation Therapeutics, 2665 East Mall, 2 Floor, Vancouver, BC V6T 1Z4, Canada.
Fariba SadaatiNanoVation Therapeutics, 2665 East Mall, 2 Floor, Vancouver, BC V6T 1Z4, Canada.
Pamela K WagnerUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.
Terri L PetkauUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.
Marco A CiufoliniNanoVation Therapeutics, 2665 East Mall, 2 Floor, Vancouver, BC V6T 1Z4, Canada.
Pieter R CullisUniversity of British Columbia, Department of Biochemistry and Molecular Biology, 2350 Health Sciences Mall, Vancouver, BC V6T 1Z3, Canada.
Blair R LeavittUniversity of British Columbia, Department of Medical Genetics, C201-4500 Oak Street, Vancouver, BC V6H 3N1, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Many genetic neurological diseases are caused by toxic gain-of-function of a mutant protein or loss-of-function of a wild-type protein. Treatment of these disorders may be feasible using gene therapy, which requires delivering therapeutic agents to affected brain regions and cells. Lipid nanoparticles (LNPs) have significant potential for this purpose: the clinical safety and efficacy of LNP systems is well-established, and neurons are amenable to LNP-mediated transfection. To adapt LNP technology for brain gene therapy applications, we iteratively designed LNPs to deliver nucleic acids to

Indexed as

brain gene therapylipid nanoparticlesMT: delivery strategiesnanomedicineneurodegenerative diseaseneurological disease

Identifiers

PMID42597608
PMCPMC13469846

What OpenQuestion holds

Textmetadata
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.