Evidence map›Paper›PMID 42662950›Full record

ArticleMolecular therapy. Nucleic acids2026

Codon-optimized

Ellen R Koufer, Alice Townsend, Youngseo Na, Adele B Correia, Noah K Latham, Koralege C Pathmasiri, Olga Ilnytska, Judith Storch, Kyle A Sullivan, Troy Halseth and 8 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

18 authors.

Ellen R KouferStead Family Department of Pediatrics, University of Iowa, Iowa City, IA 52242, USA.
Alice TownsendBredesen Center for Interdisciplinary Research and Graduate Education, University of Tennessee-Knoxville, Knoxville, TN 37996, USA.
Youngseo NaDepartment of Pharmaceutical Sciences, University of Michigan, Ann Arbor, MI 48109, USA.
Adele B CorreiaDepartment of Pathology, University of Michigan, Ann Arbor, MI 48109, USA.
Noah K LathamStead Family Department of Pediatrics, University of Iowa, Iowa City, IA 52242, USA.
Koralege C PathmasiriDepartment of Chemistry, College of Liberal Arts and Sciences, University of Illinois, Chicago, Chicago, IL 60607-7061, USA.
Olga IlnytskaDepartment of Nutritional Sciences, Rutgers University, New Brunswick, NJ 08901, USA.
Judith StorchDepartment of Nutritional Sciences, Rutgers University, New Brunswick, NJ 08901, USA.
Kyle A SullivanComputational and Predictive Biology, Oak Ridge National Laboratory, Oak Ridge, TN 37831, USA.
Troy HalsethDepartment of Pharmaceutical Sciences, University of Michigan, Ann Arbor, MI 48109, USA.
Thaddeus J KunkelDepartment of Pathology, University of Michigan, Ann Arbor, MI 48109, USA.
Elizabeth S PhillippiStead Family Department of Pediatrics, University of Iowa, Iowa City, IA 52242, USA.
Matthew L LiebermanStead Family Department of Pediatrics, University of Iowa, Iowa City, IA 52242, USA.
Stephanie M ColognaDepartment of Chemistry, College of Liberal Arts and Sciences, University of Illinois, Chicago, Chicago, IL 60607-7061, USA.
Andrew P LiebermanDepartment of Pathology, University of Michigan, Ann Arbor, MI 48109, USA.
Daniel A JacobsonComputational and Predictive Biology, Oak Ridge National Laboratory, Oak Ridge, TN 37831, USA.
Anna S SchwendemanDepartment of Pharmaceutical Sciences, University of Michigan, Ann Arbor, MI 48109, USA.
Mark L SchultzStead Family Department of Pediatrics, University of Iowa, Iowa City, IA 52242, USA.

Funding

Therapeutic Targets for Niemann-Pick Type C NeurodegenerationR01NS122746 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI LIEBERMAN, ANDREW P · 2021 to 2025
$3.0M
Predoctoral Training Program in GeneticsT32GM145441 · NIGMS · UNIVERSITY OF IOWA · PI Josep M Comeron, DANIEL F EBERL · 2022 to 2026
$1.5M
Regulation and Mechanisms of ER ProteostasisR35GM154959 · NIGMS · UNIVERSITY OF IOWA · PI Mark Louis Schultz · 2024 to 2026
$1.1M
IVIS Biolumenscence and Fluorescence Live Animal ImagingS10OD026835 · OD · UNIVERSITY OF IOWA · PI RICE, KEVIN G · 2019 to 2019
$257k
NIGMS NIH HHS R35 GM154959NIGMS NIH HHS T32 GM145441NIH HHS S10 OD026835NINDS NIH HHS R01 NS122746
6 · The paper itself

Abstract

Niemann-Pick type C1 disease is a lysosomal storage disorder caused by mutations in the

Indexed as

lipid nanoparticlelysosomal storage disordermRNA therapyMT: RNA/DNA editingNiemann-Pick type C1Niemann-Pick type C1 liverNPC1

Identifiers

PMID42662950
PMCPMC13521171

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.