ArticleEMBO molecular medicine2021
Development of a small molecule that corrects misfolding and increases secretion of Z α
Open access · goldAbstract read
In one paragraph
Article in EMBO molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed
3.4field-weighted citation impact, top 6% of its field
What it found
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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.
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Who cites it
32 citing papers in PubMed, 62 citations in OpenAlex.
- Z variant heterozygosity in alpha-1 antitrypsin deficiency: disease risk and treatment implications.Orphanet journal of rare diseases · 2026Article
- The mechanism of pathogenic αProceedings of the National Academy of Sciences of the United States of America · 2025Article
- Molecular insights on the mechanism of α1-antitrypsin condensate formation and maturation.PLoS computational biology · 2025Article
- A cure for alpha-1? Novel therapeutics in alpha-1 antitrypsin deficiency.The European respiratory journal · 2025Article
- Susceptibility of alpha-1 antitrypsin deficiency variants to polymer-blocking therapy.JCI insight · 2025Article
- Chaperone-mediated autophagy degrades SERPINA1Autophagy · 2025Article
- Top-Down Ion Mobility Mass Spectrometry Reveals a Disease Associated Conformational Ensemble of Alpha-1-antitrypsin.Journal of the American Chemical Society · 2025Article
- High-resolution characterization of ex vivo AAT polymers by solution-state NMR spectroscopy.Science advances · 2025Article
- Evaluation of DNA encoded library and machine learning model combinations for hit discovery.npj drug discovery · 2025Article
- An Inducible Neural Stem Progenitor Cell Model for Testing Therapeutic Interventions Against Neurodegeneration FENIB.Drug development research · 2025Article
- Spatial covariance reveals isothiocyanate natural products adjust redox stress to restore function in alpha-1-antitrypsin deficiency.Cell reports. Medicine · 2025Article
- Alpha-1 Antitrypsin Inclusions Sequester GRP78 in a Bile Acid-Inducible Manner.Liver international : official journal of the International Association for the Study of the Liver · 2025Article
- Making Proteins with Electricity.Reviews of physiology, biochemistry and pharmacology · 2025Review
- A novel pathological mutant reveals the role of torsional flexibility in the serpin breach in adoption of an aggregation-prone intermediate.The FEBS journal · 2024Article
- Tracing genetic diversity captures the molecular basis of misfolding disease.Nature communications · 2024Article
- Native and Ion Mobility Mass Spectrometry Characterization of Alpha 1 Antitrypsin Variants and Oligomers.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Future Perspectives in the Diagnosis and Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency.GE Portuguese journal of gastroenterology · 2023Review
- Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Small-molecule discovery through DNA-encoded libraries.Nature reviews. Drug discovery · 2023Review
- The under-appreciated world of the serpin family of serine proteinase inhibitors.EMBO molecular medicine · 2023Article
Corrections and comments
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Authors and funding
37 authors at 5 institutions in 5 countries.
David A LomasUCL Respiratory, Rayne Institute, University College London, London, UK.ORCID 0000-0003-2339-6979
James A IrvingUCL Respiratory, Rayne Institute, University College London, London, UK.ORCID 0000-0003-3204-6356
Christopher Arico-MuendelGlaxoSmithKline, Cambridge, MA, USA.
Svetlana BelyanskayaGlaxoSmithKline, Cambridge, MA, USA.
Andrew BrewsterGlaxoSmithKline, Stevenage, UK.
Murray BrownGlaxoSmithKline, Stevenage, UK.
Chun-Wa ChungGlaxoSmithKline, Stevenage, UK.
Hitesh DaveGlaxoSmithKline, Stevenage, UK.
Alexis DenisGlaxoSmithKline, Paris, France.
Nerina DodicGlaxoSmithKline, Paris, France.
Anthony DossangGlaxoSmithKline, Stevenage, UK.
Peter EddershawGlaxoSmithKline, Stevenage, UK.
Diana KlimaszewskaGlaxoSmithKline, Stevenage, UK.
Imran HaqUCL Respiratory, Rayne Institute, University College London, London, UK.
Duncan S HolmesGlaxoSmithKline, Stevenage, UK.
Jonathan P HutchinsonGlaxoSmithKline, Stevenage, UK.
Alistair M JaggerUCL Respiratory, Rayne Institute, University College London, London, UK.
Toral JakhriaGlaxoSmithKline, Stevenage, UK.
Emilie JigorelGlaxoSmithKline, Paris, France.
John LiddleGlaxoSmithKline, Stevenage, UK.
Ken LindGlaxoSmithKline, Cambridge, MA, USA.
Stefan J MarciniakCambridge Institute for Medical Research, Cambridgem, UK.
Jeff MesserGlaxoSmithKline, Cambridge, MA, USA.
Margaret NeuGlaxoSmithKline, Stevenage, UK.
Allison OlszewskiGlaxoSmithKline, Cambridge, MA, USA.
Adriana OrdonezCambridge Institute for Medical Research, Cambridgem, UK.
Riccardo RonzoniUCL Respiratory, Rayne Institute, University College London, London, UK.ORCID 0000-0002-3981-8104
James RowedderGlaxoSmithKline, Stevenage, UK.
Martin RüdigerGlaxoSmithKline, Stevenage, UK.
Steve SkinnerGlaxoSmithKline, Cambridge, MA, USA.
Kathrine J SmithGlaxoSmithKline, Stevenage, UK.
Rebecca TerryGlaxoSmithKline, Stevenage, UK.
Lionel TrottetGlaxoSmithKline, Paris, France.
Iain UingsGlaxoSmithKline, Stevenage, UK.
Steve WilsonGlaxoSmithKline, Stevenage, UK.
Zhengrong ZhuGlaxoSmithKline, Cambridge, MA, USA.
Andrew C PearceGlaxoSmithKline, Stevenage, UK.ORCID 0000-0002-4698-037X
GlaxoSmithKline (United Kingdom) · GBGlaxoSmithKline (India) · INUniversity College London · GBGlaxoSmithKline (France) · FRInstitute for Medical Research · MY
Funding
Biotechnology and Biological Sciences Research Council BB/T002603/1Department of HealthMedical Research Council G0601840Medical Research Council G1002610Medical Research Council MR/N024842/1Medical Research Council MR/R009120/1Medical Research Council MR/V028669/1Medical Research Council MR/V034243/1
Abstract
Severe α
Indexed as
alpha 1-Antitrypsinalpha 1-Antitrypsin DeficiencyAnimalsEndoplasmic ReticulumHepatocytesMicealpha 1-Antitrypsinemphysemaliver diseaseprotein misfoldingsmall molecule correctorα1-antitrypsin deficiency
Identifiers
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.