Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo.
- DOI
- 10.1016/j.omtn.2026.103022
- Published
- 2026 Sep 8
- Container
- Molecular therapy. Nucleic acids
- Publisher
- Not recorded
- Open access
- yes
Credibility signals
limited evidence Score 45/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.
Show all credibility signals
- cautionDOI registered: No matching Crossref record was present in this response.
- cautionDOI resolves: No matching Crossref record was present in this response.
- not scoredDirectory of Open Access Journals: No matching DOAJ record was present in this response. No allow-list match; this is not evidence of low credibility.
- not scoredMEDLINE indexed: Not checked or no result supplied; no credibility inference made.
- not scoredOpenAlex core source: Not checked or no result supplied; no credibility inference made.
- not scoredKnown publisher allow-list: Not checked or no result supplied; no credibility inference made.
- not scoredROR affiliation: Not checked or no result supplied; no credibility inference made.
- not scoredRetraction Watch retraction: No retraction notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete.
- not scoredRetraction Watch expression of concern: No expression of concern notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete.
- not scoredRetraction Watch correction: No correction notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete.
- not scoredRetraction Watch reinstatement: No reinstatement notice matched this DOI in the deployed snapshot. No matching event found; coverage may be incomplete.
- supportingOpen access status: Normalized open-access status: open.
- not scoredPublication license: Not checked or no result supplied; no credibility inference made.
- not scoredPublication version: A publication version was supplied but is not scored.
- cautionMetadata completeness: 5 of 6 scored descriptive metadata groups are present; missing fields increase uncertainty.
Cite this work
BibTeX
@article{allodium:10.1016/j.omtn.2026.103022,
title = {Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo.},
author = {Koufer ER and Townsend A and Na Y and Correia AB and Latham NK and Pathmasiri KC and Ilnytska O and Storch J and Sullivan KA and Halseth T and Kunkel TJ and Phillippi ES and Lieberman ML and Cologna SM and Lieberman AP and Jacobson DA and Schwendeman AS and Schultz ML},
year = {2026},
journal = {Molecular therapy. Nucleic acids},
doi = {10.1016/j.omtn.2026.103022},
url = {https://doi.org/10.1016/j.omtn.2026.103022}
}RIS
TY - JOUR TI - Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo. AU - Koufer ER AU - Townsend A AU - Na Y AU - Correia AB AU - Latham NK AU - Pathmasiri KC AU - Ilnytska O AU - Storch J AU - Sullivan KA AU - Halseth T AU - Kunkel TJ AU - Phillippi ES AU - Lieberman ML AU - Cologna SM AU - Lieberman AP AU - Jacobson DA AU - Schwendeman AS AU - Schultz ML PY - 2026 JO - Molecular therapy. Nucleic acids DO - 10.1016/j.omtn.2026.103022 UR - https://doi.org/10.1016/j.omtn.2026.103022 ER -
APA
ER, K., A, T., Y, N., AB, C., NK, L., KC, P., O, I., J, S., KA, S., T, H., TJ, K., ES, P., ML, L., SM, C., AP, L., DA, J., AS, S., & ML, S. (2026). Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo.. Molecular therapy. Nucleic acids. https://doi.org/10.1016/j.omtn.2026.103022
Source records
- pubmed · retrieved 2026-09-26T22:45:31.087Z