ArticleJournal of nucleic acids2018
SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency.
Article in Journal of nucleic acids, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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.
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.
Who cites it
31 citing papers in PubMed, 69 citations in OpenAlex.
- Delivering the blueprint: Advances and challenges in mRNA therapeutics for the respiratory system.International journal of pharmaceutics: X · 2026Review
- New approach methodologies (NAMs) for preclinical and translational evaluation of mRNA-lipid nanoparticle (LNP) therapeutics.Journal of controlled release : official journal of the Controlled Release Society · 2026Review
- Human Genetic Analysis Reveals Circulating Alpha-1 Antitrypsin Level as a Protective Factor in Sepsis.medRxiv : the preprint server for health sciences · 2026Article
- Scaled up fed-batch production of recombinant alpha-1-antitrypsin by CHO cells in single-use surface aerated orbital shaken bioreactor.Scientific reports · 2026Article
- Review
- LncRNAs, RNA Therapeutics, and Emerging Technologies in Liver Pathobiology.Seminars in liver disease · 2025Review
- Spatial covariance reveals isothiocyanate natural products adjust redox stress to restore function in alpha-1-antitrypsin deficiency.Cell reports. Medicine · 2025Article
- Alpha-1-antitrypsin-deficiency is associated with lower cardiovascular risk: an approach based on federated learning.Respiratory research · 2024Article
- Messenger RNA-Based Therapeutics and Vaccines: What's beyond COVID-19?ACS pharmacology & translational science · 2023Review
- Nanomaterials for mRNA-based therapeutics: Challenges and opportunities.Bioengineering & translational medicine · 2023Review
- Unlocking the promise of mRNA therapeutics.Nature biotechnology · 2022Review
- The legacy of mRNA engineering: A lineup of pioneers for the Nobel Prize.Molecular therapy. Nucleic acids · 2022Review
- Current Advances in RNA Therapeutics for Human Diseases.International journal of molecular sciences · 2022Review
- Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery.Journal of controlled release : official journal of the Controlled Release Society · 2022Article
- Chemically modified mRNA beyond COVID-19: Potential preventive and therapeutic applications for targeting chronic diseases.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2022Review
- mRNA therapy for myocardial infarction: A review of targets and delivery vehicles.Frontiers in bioengineering and biotechnology · 2022Review
- Advanced human-relevant in vitro pulmonary platforms for respiratory therapeutics.Advanced drug delivery reviews · 2021Review
- In Vitro Investigations on Optimizing and Nebulization of IVT-mRNA Formulations for Potential Pulmonary-Based Alpha-1-Antitrypsin Deficiency Treatment.Pharmaceutics · 2021Article
- Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety.Biomedicines · 2021Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Alpha-1-antitrypsin (AAT) deficiency is a genetic disorder that produces inactive/defective AAT due to mutations in the SERPINA1 gene encoding AAT. This disease is associated with decreased activity of AAT in the lungs and deposition of excessive defective AAT protein in the liver. Currently there is no specific treatment for liver disease associated with AAT deficiency. AAT lung disease is often treated with one of several serum protein replacement products; however, long-term studies of the effectiveness of SerpinA1 replacement therapy are not available, and it does not reduce liver damage in AAT deficiency. mRNA therapy could potentially target both the liver and lungs of AAT deficient patients. AAT patient fibroblasts and AAT patient fibroblast-derived hepatocytes were transfected with SERPINA1-encoding mRNA and cell culture media were tested for SerpinA1 expression. Our data demonstrates increased SerpinA1 protein in culture media from treated AAT patient fibroblasts and AAT patient fibroblast-derived hepatocytes. In vivo studies in wild type mice demonstrate SERPINA1 mRNA biodistribution in liver and lungs, as well as SerpinA1 protein expression in these two target organs which are critically affected in AAT deficiency. Taken together, our data suggests that SerpinA1 mRNA therapy has the potential to benefit patients suffering from AAT deficiency.
Identifiers
What OpenQuestion holds
Registered trials
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.