Evidence map›Paper›PMID 41511298›Full record

ReviewCells2025

Engineering Liver-Specific Promoters: A Comprehensive Review of Design, Mechanisms, and Clinical Applications in Gene Therapy.

Valentin Artemyev, Anastasiia Iu Paremskaia, Amina A Dzhioeva, Daria Mishina, Viktor Bogdanov, Julia Krupinova, Ali Mazloum, Sofya G Feoktistova, Olga N Mityaeva, Pavel Yu Volchkov

Abstract readReview
In one paragraph

Review in Cells, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

10 authors.

Valentin ArtemyevMoscow Center for Advanced Studies, Kulakova Str. 20, Moscow 123592, Russia.ORCID 0009-0001-1774-6656
Anastasiia Iu ParemskaiaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0009-0008-0986-686X
Amina A DzhioevaMoscow Center for Advanced Studies, Kulakova Str. 20, Moscow 123592, Russia.ORCID 0009-0000-7239-8474
Daria MishinaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0009-0002-7188-6956
Viktor BogdanovFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0001-6377-9056
Julia KrupinovaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0001-7963-5022
Ali MazloumFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0002-5982-8393
Sofya G FeoktistovaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0002-7608-439X
Olga N MityaevaFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0001-9252-1601
Pavel Yu VolchkovFederal Research Center for Innovator and Emerging Biomedical and Pharmaceutical Technologies, Moscow 125315, Russia.ORCID 0000-0001-9794-6297

Funding

Ministry of Science and Higher Education of the Russian Federation 075-03-2023-106/12Russian Science Foundation 23-64-00002
6 · The paper itself

Abstract

The liver is a primary metabolic hub and a pivotal target for gene therapy, owing to its capacity for protein secretion, role in metabolic homeostasis and immune tolerance. Liver-directed gene therapies are used to treat numerous inherited metabolic disorders and coagulation factor deficiencies including hemophilia (A and B), Crigler-Najjar syndrome, mucopolysaccharidoses, phenylketonuria, Fabry, Gaucher, Wilson and Pompe diseases. The efficacy and safety of liver-directed gene therapy rely on the use of strong tissue-specific promoters. To date, there are many different liver-specific promoters used in preclinical and clinical studies, including novel completely synthetic promoters. This review provides a comprehensive analysis of the design, engineering and application of liver-specific promoters. Furthermore, we discuss fundamental principles of gene expression regulation in the liver and the physiological and immunological characteristics that make it a suitable target organ for gene therapy delivery.

Indexed as

Genetic EngineeringGenetic TherapyLiverPromoter Regions, GeneticAnimalsGene Expression RegulationGene Therapy AgentsGenetic VectorsHumansgene regulationgene therapyimmune privilegeliver-specific promoterspromoters designsynthetic promotersviral vectors

Identifiers

PMID41511298
PMCPMC12786076

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.