Evidence map›Paper›PMID 38585818›Full record

ArticlebioRxiv : the preprint server for biology2024

Glycoengineered recombinant alpha1-antitrypsin results in comparable

Frances Rocamora, Sanne Schoffelen, Johnny Arnsdorf, Eric A Toth, Yunus Abdul, Thomas E Cleveland, Sara Petersen Bjørn, Mina Ying Min Wu, Noel G McElvaney, Bjørn Gunnar Rude Voldborg and 2 more

Open access · greenAbstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2024. 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, 1 citations in OpenAlex.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 6 institutions in 4 countries.

Frances RocamoraDepartment of Pediatrics, University of California, San Diego, School of Medicine, La Jolla, CA 92093, United States.ORCID 0000-0002-2579-6733
Sanne SchoffelenNational Biologics Facility, Department of Biotechnology and Biomedicine, Technical University of Denmark, Lyngby, Denmark.ORCID 0000-0003-2664-8561
Johnny ArnsdorfNational Biologics Facility, Department of Biotechnology and Biomedicine, Technical University of Denmark, Lyngby, Denmark.ORCID 0000-0002-2738-0811
Eric A TothInstitute for Bioscience and Biotechnology Research, University of Maryland, Rockville, MD 20850, United States.
Yunus AbdulInstitute for Bioscience and Biotechnology Research, University of Maryland, Rockville, MD 20850, United States.
Thomas E ClevelandNational Institute of Standards and Technology, Rockville, MD, 20850, USA.
Sara Petersen BjørnNational Biologics Facility, Department of Biotechnology and Biomedicine, Technical University of Denmark, Lyngby, Denmark.ORCID 0000-0003-3341-6160
Mina Ying Min WuDepartment of Pediatrics, University of California, San Diego, School of Medicine, La Jolla, CA 92093, United States.
Noel G McElvaneyDepartment of Bioengineering, University of California, San Diego, La Jolla, CA 92093, United States.ORCID 0000-0002-0152-4370
Bjørn Gunnar Rude VoldborgNational Biologics Facility, Department of Biotechnology and Biomedicine, Technical University of Denmark, Lyngby, Denmark.ORCID 0000-0002-7005-1642
Thomas R FuerstInstitute for Bioscience and Biotechnology Research, University of Maryland, Rockville, MD 20850, United States.
Nathan E LewisDepartment of Pediatrics, University of California, San Diego, School of Medicine, La Jolla, CA 92093, United States.ORCID 0000-0001-7700-3654
Technical University of Denmark · DKUniversity of California San Diego · USAdvanced Bioscience Laboratories (United States) · USNational Institute of Standards and Technology · USNexImmune (United States) · USRoyal College of Surgeons in Ireland · IE

Funding

Glycoengineering of CHO cells to express recombinant alpha-1 antitrypsinR41HL164260 · NHLBI · NEUIMMUNE BIOLOGICS, INC. · PI LEWIS, NATHAN ENOCH · 2022 to 2022
$274k
NHLBI NIH HHS R41 HL164260
6 · The paper itself

Abstract

Alpha-1-antitrypsin (A1AT) is a multifunctional, clinically important, high value therapeutic glycoprotein that can be used for the treatment of many diseases such as alpha-1-antitrypsin deficiency, diabetes, graft-versus-host-disease, cystic fibrosis and various viral infections. Currently, the only FDA-approved treatment for A1AT disorders is intravenous augmentation therapy with human plasma-derived A1AT. In addition to its limited supply, this approach poses a risk of infection transmission, since it uses therapeutic A1AT harvested from donors. To address these issues, we sought to generate recombinant human A1AT (rhA1AT) that is chemically and biologically indistinguishable from its plasma-derived counterpart using glycoengineered Chinese Hamster Ovary (geCHO-L) cells. By deleting nine key genes that are part of the CHO glycosylation machinery and expressing the human ST6GAL1 and A1AT genes, we obtained stable, high producing geCHO-L lines that produced rhA1AT having an identical glycoprofile to plasma-derived A1AT (pdA1AT). Additionally, the rhA1AT demonstrated

Indexed as

alpha-1-antitrypsinbiologicglycoengineeringglycosylationtherapeutic protein

Identifiers

PMID38585818
PMCPMC10996670
OpenAlexW4393338856

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.